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2-Methylbutyryl-CoA dehydrogenase deficiency Other names2-Methylbutyryl glycinuria or Short/branched-chain acyl-CoA dehydrogenase deficiency (SBCADD),[1] 2-Methylbutyryl-CoA 2-Methylbutyryl-CoA dehydrogenase deficiency, is an autosomal recessive metabolic disorder.[2] It causes the body to be unable to p...
2-Methylbutyryl-CoA dehydrogenase deficiency
c1864912
400
wikipedia
https://en.wikipedia.org/wiki/2-Methylbutyryl-CoA_dehydrogenase_deficiency
2021-01-18T18:29:53
{"gard": ["10322"], "mesh": ["C566487"], "umls": ["C1864912"], "orphanet": ["79157"], "wikidata": ["Q4596905"]}
Ring chromosome 16 is characterized by postnatal growth deficiency, intellectual disability, microcephaly, broad flat nasal bridge, down-turned mouth, low-set and dysmorphic (abnormally-shaped) ears and speech delay. To date, less than 10 cases have been reported in the medical literature. *[v]: View this temp...
Ring chromosome 16
c4706449
401
gard
https://rarediseases.info.nih.gov/diseases/10855/ring-chromosome-16
2021-01-18T17:57:53
{"orphanet": ["96178"], "synonyms": ["Ring chromosome 16", "Ring 16", "Chromosome 16 ring", "R16"]}
Extramammary Paget disease is a cancerous disease seen at various sites, most often in the anogenital region. The clinical features are usually those of eczematous eruptions with weeping and crust formation. This disease has been shown to be a skin manifestation of internal malignancy. In a study of 40 patients with ...
PAGET DISEASE, EXTRAMAMMARY
c0030186
402
omim
https://www.omim.org/entry/167300
2019-09-22T16:36:46
{"mesh": ["D010145"], "omim": ["167300"], "orphanet": ["2800"]}
A group of disorders characterized by congenital limb contractures manifesting as limitation of movement of multiple limb joints at birth that is usually non-progressive and may include muscle weakness and fibrosis. This disorder is always associated with decreased intrauterine fetal movement which leads secondarily ...
Arthrogryposis multiplex congenita
c0003886
403
orphanet
https://www.orpha.net/consor/cgi-bin/OC_Exp.php?lng=EN&Expert=1037
2021-01-23T17:43:21
{"gard": ["777"], "mesh": ["C536613", "D001176"], "umls": ["C2931264"], "icd-10": ["Q74.3"], "synonyms": ["AMC", "Multiple congenital arthrogryposis"]}
## Clinical Features Huson et al. (1993) described what they considered to be a previously unrecognized form of autosomal recessive spondyloepiphyseal dysplasia tarda in 3 sibs, the offspring of first-cousin Pakistani parents. In addition to the skeletal dysplasia, microcephaly, unusual facies, and moderate develop...
SPONDYLOEPIPHYSEAL DYSPLASIA TARDA WITH CHARACTERISTIC FACIES
c1838653
404
omim
https://www.omim.org/entry/600093
2019-09-22T16:16:42
{"mesh": ["C564003"], "omim": ["600093"]}
A rare syndrome with combined immunodeficiency characterized by a variable clinical presentation ranging from asymptomatic individuals to potentially life-threatening, recurrent bacterial infections associated with progressive loss of serum immunoglobulins and B cells. *[v]: View this template *[t]: Discuss this...
Pancytopenia due to IKZF1 mutations
c4225173
405
orphanet
https://www.orpha.net/consor/cgi-bin/OC_Exp.php?lng=EN&Expert=317473
2021-01-23T17:59:29
{"omim": ["616873"], "icd-10": ["D81.8"], "synonyms": ["CID due to IKAROS deficiency", "Combined immunodeficiency due to IKAROS deficiency"]}
Steroid dementia syndrome SpecialtyNeurology/psychiatry Steroid dementia syndrome describes the signs and symptoms of hippocampal and prefrontal cortical dysfunction, such as deficits in memory, attention, and executive function, induced by glucocorticoids.[1] Dementia-like symptoms have been found in some ind...
Steroid dementia syndrome
None
406
wikipedia
https://en.wikipedia.org/wiki/Steroid_dementia_syndrome
2021-01-18T18:33:28
{"wikidata": ["Q17155309"]}
A number sign (#) is used with this entry because of evidence that this Marfan-like syndrome can be caused by mutation in the gene encoding fibrillin (FBN1; 134797) on chromosome 15q21.1. Clinical Features Glesby and Pyeritz (1989) pointed out that more than half of a large number of patients evaluated in the m...
MASS SYNDROME
c1858556
407
omim
https://www.omim.org/entry/604308
2019-09-22T16:12:13
{"mesh": ["C536030"], "omim": ["604308"], "orphanet": ["99715"], "synonyms": ["Alternative titles", "Mitral valve-aorta-skeleton-skin syndrome", "MASS PHENOTYPE", "OVERLAP CONNECTIVE TISSUE DISEASE"]}
Human disease Adult T-cell leukemia/lymphoma Human T-cell(normal) SpecialtyOncology, hematology Adult T-cell leukemia/lymphoma (ATL or ATLL) is a rare cancer of the immune system's T-cells[1][2][3] caused by human T cell leukemia/lymphotropic virus type 1 (HTLV-1).[4] ## Contents * 1 Signs and symptoms ...
Adult T-cell leukemia/lymphoma
c0023493
408
wikipedia
https://en.wikipedia.org/wiki/Adult_T-cell_leukemia/lymphoma
2021-01-18T18:37:09
{"gard": ["13103"], "mesh": ["D015459"], "umls": ["C0023493"], "icd-9": ["204.0", "208.9"], "orphanet": ["86875"], "wikidata": ["Q379054"]}
A number sign (#) is used with this entry because 15 to 20% of cases of familial amyotrophic lateral sclerosis (FALS), referred to here as ALS1, are associated with mutations in the superoxide dismutase-1 gene (SOD1; 147450) on chromosome 21q22. Although most cases of SOD1-related familial ALS follow autosomal do...
AMYOTROPHIC LATERAL SCLEROSIS 1
c0002736
409
omim
https://www.omim.org/entry/105400
2019-09-22T16:45:13
{"doid": ["0060193"], "mesh": ["D000690"], "omim": ["105400"], "orphanet": ["803"], "synonyms": ["Alternative titles", "AMYOTROPHIC LATERAL SCLEROSIS 1, FAMILIAL", "AMYOTROPHIC LATERAL SCLEROSIS 1, AUTOSOMAL DOMINANT"], "genereviews": ["NBK1450"]}
A number sign (#) is used with this entry because primary pigmented nodular adrenocortical disease-1 (PPNAD1) is caused by heterozygous mutation in the protein kinase A regulatory subunit 1-alpha gene (PRKAR1A; 188830) on chromosome 17q. Description Primary pigmented micronodular adrenocortical disease is a form of...
PIGMENTED NODULAR ADRENOCORTICAL DISEASE, PRIMARY, 1
c1864846
410
omim
https://www.omim.org/entry/610489
2019-09-22T16:04:26
{"doid": ["0060280"], "mesh": ["C566469"], "omim": ["610489"], "orphanet": ["189439"], "synonyms": ["CUSHING SYNDROME, ADRENAL, DUE TO PPNAD1", "Primary pigmented nodular adrenal dysplasia", "PPNAD", "Alternative titles", "PIGMENTED MICRONODULAR ADRENOCORTICAL DISEASE, PRIMARY, 1", "ADRENOCORTICAL NODULAR DYSPLASIA, PR...
Colitis X, equine colitis X or peracute toxemic colitis is a catchall term for various fatal forms of acute or peracute colitis found in horses, but particularly a fulminant colitis where clinical signs include sudden onset of severe diarrhea, abdominal pain, shock, and dehydration. Death is common, with 90% to 100% ...
Colitis-X
c0267550
411
wikipedia
https://en.wikipedia.org/wiki/Colitis-X
2021-01-18T18:55:38
{"wikidata": ["Q5145774"]}
This article is about humans. For other mammals, see pseudopregnancy. False pregnancy Other namesPhantom pregnancy, hysterical pregnancy, pseudocyesis, delusional pregnancy SpecialtyPsychiatry False pregnancy (or pseudocyesis, from the Greek pseudes "false" and kyesis "pregnancy"[1]) is the appearance of cl...
False pregnancy
c1827755
412
wikipedia
https://en.wikipedia.org/wiki/False_pregnancy
2021-01-18T19:01:49
{"mesh": ["D011555"], "icd-9": ["300.11"], "icd-10": ["F45.8"], "wikidata": ["Q1065846"]}
Late-onset focal dermal elastosis is a rare, acquired, dermis elastic tissue disorder characterized by a pseudoxanthoma elasticum-like papular eruption consisting of multiple, slowly progressive, asymptomatic, 2-5 mm, white to yellowish, non-follicular papules (that tend to form cobblestone plaques) predominantly...
Late-onset focal dermal elastosis
None
413
orphanet
https://www.orpha.net/consor/cgi-bin/OC_Exp.php?lng=EN&Expert=228227
2021-01-23T18:15:08
{"synonyms": ["PXE-like late-onset focal dermal elastosis", "Pseudoxanthoma-like late-onset focal dermal elastosis"]}
Meningiomas are tumors that originate in the meninges, the membranes that surround the brain and spinal cord. Most meningiomas are not cancerous (benign), though a minority of meningiomas can be classified as atypical or cancerous (malignant). Though rare, malignant meningiomas can be highly aggressive. However, even...
Meningioma
c0025286
414
gard
https://rarediseases.info.nih.gov/diseases/7015/meningioma
2021-01-18T17:59:09
{"mesh": ["D008579"], "orphanet": ["2495"], "synonyms": []}
A number sign (#) is used with this entry because of evidence that spermatogenic failure-14 (SPGF14) is caused by homozygous mutation in the ZMYND15 gene (614312) on chromosome 17p13. One such family has been reported. For a general phenotypic description and a discussion of genetic heterogeneity of spermatogenic fa...
SPERMATOGENIC FAILURE 14
c4014454
415
omim
https://www.omim.org/entry/615842
2019-09-22T15:50:50
{"doid": ["0070179"], "omim": ["615842"], "orphanet": ["399805"], "synonyms": []}
A rare hereditary ataxia characterized by neurogenic muscular atrophy associated with signs of cerebellar ataxia, hypesthesia, degeneration of the retina, and diabetes mellitus. Onset of the disease is in adolescence and the course is slowly progressive. There have been no further descriptions in the literature since...
Muscular atrophy-ataxia-retinitis pigmentosa-diabetes mellitus syndrome
c0342281
416
orphanet
https://www.orpha.net/consor/cgi-bin/OC_Exp.php?lng=EN&Expert=2579
2021-01-23T17:55:38
{"gard": ["2417"], "mesh": ["C562774", "C538193"], "omim": ["158500"], "umls": ["C0342281", "C2931765"], "icd-10": ["G11.0"], "synonyms": ["Furukawa-Takagi-Nakao syndrome"]}
A number sign (#) is used with this entry because of evidence that Pilarowski-Bjornsson syndrome (PILBOS) is caused by heterozygous mutation in the CHD1 gene (602118) on chromosome 5q. Description Pilarowski-Bjornsson syndrome is an autosomal dominant neurodevelopmental disorder characterized by delayed development...
PILAROWSKI-BJORNSSON SYNDROME
c4540131
417
omim
https://www.omim.org/entry/617682
2019-09-22T15:45:11
{"omim": ["617682"], "orphanet": ["529965"], "synonyms": ["Pilarowski-Bjornsson syndrome", "Alternative titles", "DEVELOPMENTAL DELAY AND SPEECH APRAXIA WITH OR WITHOUT SEIZURES"]}
Disease caused by nematodes of the genus Capillaria Capillariasis is a disease caused by nematodes in the genus Capillaria.[1] The two principal forms of the disease are: * Intestinal capillariasis, caused by Capillaria philippinensis * Hepatic capillariasis, caused by Capillaria hepatica ## References[edit] ...
Capillariasis
c0006897
418
wikipedia
https://en.wikipedia.org/wiki/Capillariasis
2021-01-18T18:35:43
{"mesh": ["D017189"], "umls": ["C0006897"], "wikidata": ["Q1034620"]}
For a general phenotypic description and a discussion of genetic heterogeneity of the punctate type of palmoplantar keratoderma, see PPKP1A (148600). Clinical Features Zhang et al. (2004) studied 2 unrelated Chinese families segregating autosomal dominant punctate palmoplantar keratoderma. In the first family, ...
PALMOPLANTAR KERATODERMA, PUNCTATE TYPE IB
c3554145
419
omim
https://www.omim.org/entry/614936
2019-09-22T15:53:42
{"omim": ["614936"], "orphanet": ["79501"], "synonyms": ["Buschke-Fischer-Brauer syndrome", "Keratodermia palmoplantaris papulosa, Buschke-Fischer-Brauer type", "PPKP1"]}
A number sign (#) is used with this entry because of evidence that Blau syndrome (BLAUS) is caused by heterozygous mutation in the NOD2/CARD15 gene (605956) on chromosome 16q12. Description Blau syndrome is characterized by the triad of granulomatous arthritis, uveitis, and dermatitis. First described in 1985, it w...
BLAU SYNDROME
c1861303
420
omim
https://www.omim.org/entry/186580
2019-09-22T16:32:54
{"doid": ["0050678"], "mesh": ["C538157"], "omim": ["186580"], "icd-10": ["M04.8"], "orphanet": ["90340", "90341"], "synonyms": ["Alternative titles", "SARCOIDOSIS, EARLY-ONSET", "GRANULOMATOSIS, FAMILIAL JUVENILE SYSTEMIC", "ARTHROCUTANEOUVEAL GRANULOMATOSIS", "JABS SYNDROME", "GRANULOMATOUS INFLAMMATORY ARTHRITIS, DE...
Hyperinsulinism due to HNF1A deficiency is a form of diazoxide-sensitive diffuse hyperinsulinism (DHI), characterized by transient or persistent hyperinsulinemic hypoglycemia (HH) in infancy that is responsive to diazoxide, evolving in to maturity-onset diabetes of the young subtype 1 (MODY-1; see this term) later in...
Hyperinsulinism due to HNF1A deficiency
c4303475
421
orphanet
https://www.orpha.net/consor/cgi-bin/OC_Exp.php?lng=EN&Expert=324575
2021-01-23T17:21:19
{"icd-10": ["E16.1"], "synonyms": ["Hyperinsulinemic hypoglycemia due to HNF1A deficiency"]}
Wells syndrome is characterised by the presence of recurrent cellulitis-like eruptions with eosinophilia. ## Epidemiology Approximately 80 cases have been described in the literature. ## Clinical description The lesions are usually filled with fluid, tender and progress to form hardened plaques of edema and eryth...
Wells syndrome
c0343101
422
orphanet
https://www.orpha.net/consor/cgi-bin/OC_Exp.php?lng=EN&Expert=901
2021-01-23T18:47:05
{"gard": ["329"], "mesh": ["C536693"], "umls": ["C0343101"], "icd-10": ["L98.3"], "synonyms": ["Eosinophilic cellulitis"]}
See also: Agnosia Akinetopsia (Greek: a for "without", kine for "to move" and opsia for "seeing"), also known as cerebral akinetopsia or motion blindness, is a neuropsychological disorder in which a patient cannot perceive motion in their visual field, despite being able to see stationary objects without issue.[1] T...
Akinetopsia
None
423
wikipedia
https://en.wikipedia.org/wiki/Akinetopsia
2021-01-18T19:10:17
{"wikidata": ["Q784564"]}
Thought insertion is defined by the ICD-10 as feeling as if one's thoughts are not one's own, but rather belong to someone else and have been inserted into one's mind.[1][2][3][4] The person experiencing thought insertion will not necessarily know where the thought is coming from, but makes a distinction between thei...
Thought insertion
c0233687
424
wikipedia
https://en.wikipedia.org/wiki/Thought_insertion
2021-01-18T18:38:29
{"wikidata": ["Q7796994"]}
Posterior amorphous corneal dystrophy (PACD) is a very rare form of stromal corneal dystrophy (see this term) characterized by irregular amorphous sheet-like opacities in the posterior corneal stroma and in Descemet membrane and mildly impaired vision. ## Epidemiology Prevalence of this form of corneal dystrophy is...
Posterior amorphous corneal dystrophy
c2748502
425
orphanet
https://www.orpha.net/consor/cgi-bin/OC_Exp.php?lng=EN&Expert=98971
2021-01-23T18:10:53
{"mesh": ["C567546"], "omim": ["612868"], "umls": ["C2748502"], "icd-10": ["H18.5"], "synonyms": ["PACD", "Posterior amorphous stromal dystrophy"]}
Aphthous stomatitis Other namesRecurrent aphthous stomatitis, recurring oral aphthae, recurrent aphthous ulceration Canker sore on the lower lip SpecialtyOral medicine, dermatology Frequency~20% of people to some degree[1] Aphthous stomatitis is a common condition characterized by the repeated formation ...
Aphthous stomatitis
c0038363
426
wikipedia
https://en.wikipedia.org/wiki/Aphthous_stomatitis
2021-01-18T18:43:24
{"gard": ["5834"], "mesh": ["D013281"], "umls": ["C0038363"], "wikidata": ["Q189956"]}
For a general phenotypic description and a discussion of genetic heterogeneity of autosomal dominant spastic paraplegia, see SPG3A (182600). Clinical Features Zhao et al. (2008) reported a 4-generation Chinese family in which 7 individuals had pure spastic paraplegia inherited in an autosomal dominant pattern. ...
SPASTIC PARAPLEGIA 41, AUTOSOMAL DOMINANT
c3888208
427
omim
https://www.omim.org/entry/613364
2019-09-22T15:58:52
{"doid": ["0110793"], "omim": ["613364"], "orphanet": ["320355"], "synonyms": ["SPG41"]}
GRIN2B related syndrome is a genetic disorder that causes developmental delay and intellectual disability. Intellectual disability ranges from mild to severe. Other symptoms may include low muscle tone (hypotonia), seizures, behavior problems including autistic features, abnormal movements, a very small head (microce...
GRIN2B related syndrome
c3151411
428
gard
https://rarediseases.info.nih.gov/diseases/12851/grin2b-related-syndrome
2021-01-18T18:00:11
{"omim": ["613970"], "synonyms": ["GRIN2B-related intellectual disability", "Autosomal dominant intellectual disability-6", "GRIN2B encephalopathy", "Intellectual disability, autosomal dominant 6, with or without seizures", "GRIN2B-related neurodevelopmental disorder"]}
Barth syndrome is a metabolic and neuromuscular disorder, occurring almost exclusively in males, that primarily affects the heart, immune system, muscles, and growth. It typically becomes apparent during infancy or early childhood, but the age of onset, associated symptoms and findings, and disease course varies cons...
Barth syndrome
c0574083
429
gard
https://rarediseases.info.nih.gov/diseases/5890/barth-syndrome
2021-01-18T18:01:52
{"mesh": ["D056889"], "omim": ["302060"], "umls": ["C0574083"], "orphanet": ["111"], "synonyms": ["BTHS", "3-methylglutaconic aciduria type II", "MGA type II", "Cardioskeletal myopathy with neutropenia and abnormal mitochondria", "TAZ defect"]}
A very rare glial neoplasm of the central nervous system, most often with an intra-axial peripheral supratentorial location in one hemisphere of the frontal or parietal lobes and usually presenting in infants and young adults with symptoms of vomiting, loss of consciousness, epileptic seizures and headaches. *[v]:...
Astroblastoma
c0334587
430
orphanet
https://www.orpha.net/consor/cgi-bin/OC_Exp.php?lng=EN&Expert=251679
2021-01-23T17:14:10
{"gard": ["10635"], "mesh": ["D018302"], "umls": ["C0334587"], "icd-10": ["C71.9"]}
Timothy syndrome SpecialtyNeurology Timothy syndrome is a rare autosomal-dominant disorder characterized by physical malformations, as well as neurological and developmental defects, including heart QT-prolongation, heart arrhythmias, structural heart defects, syndactyly (webbing of fingers and toes), and auti...
Timothy syndrome
c1832916
431
wikipedia
https://en.wikipedia.org/wiki/Timothy_syndrome
2021-01-18T19:01:18
{"gard": ["9294"], "mesh": ["C536962"], "umls": ["C1832916"], "orphanet": ["768", "65283"], "wikidata": ["Q3508705"]}
## Description The Ehlers-Danlos syndrome shows phenotypic and genetic heterogeneity; see 130000. Marked joint hyperextensibility without skeletal deformity dominates the clinical picture of hypermobility-type EDS. Skin manifestations are relatively inconspicuous. Differentiation from familial joint laxity (147...
EHLERS-DANLOS SYNDROME, HYPERMOBILITY TYPE
c0268337
432
omim
https://www.omim.org/entry/130020
2019-09-22T16:41:48
{"doid": ["14757"], "mesh": ["C536196"], "omim": ["130020"], "orphanet": ["285"], "synonyms": ["Alternative titles", "EHLERS-DANLOS SYNDROME, TYPE III", "EDS III", "BENIGN HYPERMOBILITY SYNDROME"], "genereviews": ["NBK1279"]}
Cyclic vomiting syndrome is a disorder that causes recurrent episodes of nausea, vomiting, and tiredness (lethargy). This condition is diagnosed most often in young children, but it can affect people of any age. The episodes of nausea, vomiting, and lethargy last anywhere from an hour to 10 days. An affected person ...
Cyclic vomiting syndrome
c0152164
433
medlineplus
https://medlineplus.gov/genetics/condition/cyclic-vomiting-syndrome/
2021-01-27T08:24:50
{"gard": ["6230"], "mesh": ["C536228"], "omim": ["500007"], "synonyms": []}
Hypermethioninemia is an excess of a particular protein building block (amino acid), called methionine, in the blood. This condition can occur when methionine is not broken down (metabolized) properly in the body. People with hypermethioninemia often do not show any symptoms. Some individuals with hypermethioninemia...
Hypermethioninemia
c1847720
434
medlineplus
https://medlineplus.gov/genetics/condition/hypermethioninemia/
2021-01-27T08:25:27
{"gard": ["10764", "13177", "8397"], "mesh": ["C564683"], "omim": ["606664", "613752", "250850"], "synonyms": []}
A rare, genetic disorder of urea cycle metabolism characterized by either a neonatal-onset with manifestations of lethargy, poor feeding, vomiting and tachypnea or, more commonly, presentations in infancy, childhood or adulthood with chronic neurocognitive deficits, acute encephalopathy and/or coagulation defects or ...
Hyperornithinemia-hyperammonemia-homocitrullinuria syndrome
c0268540
435
orphanet
https://www.orpha.net/consor/cgi-bin/OC_Exp.php?lng=EN&Expert=415
2021-01-23T18:01:34
{"gard": ["2830"], "mesh": ["C538380"], "omim": ["238970"], "umls": ["C0268540"], "icd-10": ["E72.4"], "synonyms": ["HHH syndrome", "ORNT1 deficiency", "Ornithine carrier deficiency", "Ornithine translocase deficiency", "Triple H syndrome"]}
Arthus reaction SpecialtyEmergency medicine In immunology, the Arthus reaction (/ˌɑːrˈtjuːs/) is a type of local type III hypersensitivity reaction. Type III hypersensitivity reactions are immune complex-mediated, and involve the deposition of antigen/antibody complexes mainly in the vascular walls, serosa (pl...
Arthus reaction
c0003907
436
wikipedia
https://en.wikipedia.org/wiki/Arthus_reaction
2021-01-18T18:46:25
{"mesh": ["D001183"], "umls": ["C0003907"], "wikidata": ["Q712320"]}
Periventricular leukomalacia (PVL) is a brain injury disorder characterized by the death of the white matter of the brain due to softening of the brain tissue. It can affect fetuses or newborns, and premature babies are at the greatest risk of the disorder. PVL is caused by a lack of oxygen or blood flow to the a...
Periventricular leukomalacia
c0023529
437
gard
https://rarediseases.info.nih.gov/diseases/10285/periventricular-leukomalacia
2021-01-18T17:58:22
{"mesh": ["D007969"], "umls": ["C0023529"], "orphanet": ["171676"], "synonyms": ["PVL"]}
A number sign (#) is used with this entry because Seckel syndrome-5 is caused by homozygous or compound heterozygous mutation in the CEP152 gene (613529) on chromosome 15q21. Description Seckel syndrome is an autosomal recessive disorder characterized by proportionate short stature, severe microcephaly, mental reta...
SECKEL SYNDROME 5
c0265202
438
omim
https://www.omim.org/entry/613823
2019-09-22T15:57:18
{"doid": ["0070012"], "omim": ["613823"], "orphanet": ["808"]}
A number sign (#) is used with this entry because of evidence that familial hyperinsulinemic hypoglycemia-4 is (HHF4) is caused by homozygous mutation in the gene encoding 3-hydroxyacyl-CoA dehydrogenase (HADH; 601609) on chromosome 4q25. For a phenotypic description and discussion of genetic heterogeneity of famili...
HYPERINSULINEMIC HYPOGLYCEMIA, FAMILIAL, 4
c1291230
439
omim
https://www.omim.org/entry/609975
2019-09-22T16:05:17
{"doid": ["0070215"], "mesh": ["C535310"], "omim": ["609975"], "orphanet": ["71212"], "genereviews": ["NBK1375"]}
## Clinical Features Neubauer et al. (2006) reported a 3-generation family from northern Germany in which 4 males had a disorder characterized by early-onset leukoencephalopathy associated with metaphyseal chondrodysplasia. Inheritance was consistent with an X-linked recessive pattern. The proband developed normall...
LEUKOENCEPHALOPATHY WITH METAPHYSEAL CHONDRODYSPLASIA
c1970840
440
omim
https://www.omim.org/entry/300660
2019-09-22T16:19:50
{"mesh": ["C567065"], "omim": ["300660"], "orphanet": ["83629"]}
A pseudohallucination (from Ancient Greek: ψευδής (pseudḗs) "false, lying" + "hallucination") is an involuntary sensory experience vivid enough to be regarded as a hallucination, but considered by the person as subjective and unreal, unlike "true" hallucinations, which are considered real by patients with psychologic...
Pseudohallucination
c0233758
441
wikipedia
https://en.wikipedia.org/wiki/Pseudohallucination
2021-01-18T19:08:57
{"umls": ["C0233758"], "icd-9": ["780.1"], "wikidata": ["Q2361468"]}
Infantile acropustulosis Other namesAcropustulosis of infancy[1]:483) SpecialtyDermatology Infantile acropustulosis is an intensely itchy vesicopustular eruption of the hands and feet.[2]:205 Involvement of scabies has been suggested.[3]infantile acropustulosis is characterized by itchy papules and vesicles...
Infantile acropustulosis
c0406787
442
wikipedia
https://en.wikipedia.org/wiki/Infantile_acropustulosis
2021-01-18T18:29:04
{"umls": ["C0406787"], "wikidata": ["Q6029033"]}
In the son of healthy, consanguineous Filipino parents, Marles and Chudley (1990) described congenital absence of the ulnars with oligodactyly and endocardial fibroelastosis. The baby showed hydrops fetalis and died soon after birth. Because of the presence of 2 major malformations and the parental consanguinity,...
ULNAR AGENESIS AND ENDOCARDIAL FIBROELASTOSIS
c1848649
443
omim
https://www.omim.org/entry/276822
2019-09-22T16:21:26
{"mesh": ["C564756"], "omim": ["276822"]}
This article needs additional citations for verification. Please help improve this article by adding citations to reliable sources. Unsourced material may be challenged and removed. Find sources: "Pseudoporphyria" – news · newspapers · books · scholar · JSTOR (December 2008) (Learn how and when to remove this templ...
Pseudoporphyria
c0521616
444
wikipedia
https://en.wikipedia.org/wiki/Pseudoporphyria
2021-01-18T18:45:37
{"umls": ["C0521616"], "icd-10": ["E80.2"], "wikidata": ["Q2246688"]}
A number sign (#) is used with this entry because of evidence that peroxisomal fatty acyl-CoA reductase-1 disorder (PFCRD) is caused by homozygous or compound heterozygous mutation in the FAR1 gene (616107) on chromosome 11p15. Description Peroxisomal fatty acyl-CoA reductase-1 disorder is an autosomal recessiv...
PEROXISOMAL FATTY ACYL-CoA REDUCTASE 1 DISORDER
c4015344
445
omim
https://www.omim.org/entry/616154
2019-09-22T15:49:45
{"omim": ["616154"], "orphanet": ["438178"], "synonyms": ["Severe intellectual disability-epilepsy-cataract syndrome due to FAR1 deficiency", "Severe intellectual disability-epilepsy-cataract syndrome due to peroxisomal disorder"]}
A number sign (#) is used with this entry because mitochondrial DNA depletion syndrome-5 (MTDPS5) is caused by homozygous or compound heterozygous mutation in the beta subunit of the succinate-CoA ligase gene (SUCLA2; 603921) on chromosome 13q14. See MTDPS9 (245400) for a description of a similar disorder caused by ...
MITOCHONDRIAL DNA DEPLETION SYNDROME 5 (ENCEPHALOMYOPATHIC WITH OR WITHOUT METHYLMALONIC ACIDURIA)
c2749864
446
omim
https://www.omim.org/entry/612073
2019-09-22T16:02:22
{"doid": ["0080124"], "omim": ["612073"], "orphanet": ["254803", "1933"], "synonyms": ["mtDNA depletion syndrome, encephalomyopathic form", "mtDNA depletion syndrome, encephalomyopathic form with methylmalonic aciduria", "MITOCHONDRIAL DNA DEPLETION SYNDROME, ENCEPHALOMYOPATHIC FORM, WITH OR WITHOUT METHYLMALONIC ACIDU...
A number sign (#) is used with this entry because of evidence that hypotrichosis-13 (HYPT13) is caused by heterozygous mutation in the KRT71 gene (608245) on chromosome 12q13. One such family has been reported. For a discussion of genetic heterogeneity of hypotrichosis, see HYPT1 (605389). Clinical Features Fujimo...
HYPOTRICHOSIS 13
c0343073
447
omim
https://www.omim.org/entry/615896
2019-09-22T15:50:37
{"doid": ["0110710"], "omim": ["615896"], "orphanet": ["170"], "synonyms": ["Alternative titles", "HYPOTRICHOSIS WITH WOOLLY HAIR"]}
This article is about the replitilian disease. For other diseases, see Charcot disease. Spinal osteoarthropathy (also known as Charcot's disease) is a rare disease affecting reptiles (including snakes[1] and lizards) which causes abnormal bone growth on vertebrae, giving the reptile a lumpy appearance. The growth of...
Spinal osteoarthropathy
None
448
wikipedia
https://en.wikipedia.org/wiki/Spinal_osteoarthropathy
2021-01-18T18:43:04
{"wikidata": ["Q7577471"]}
Mitochondrial DNA depletion syndrome, encephalomyopathic form with methylmalonic aciduria (MTDPS9) affects the brain, nervous system, and muscles. Symptoms are different from person to person. They may include muscle weakness, hearing loss, and difficulty feeding and breathing. Other symptoms may include severe low m...
Mitochondrial DNA depletion syndrome, encephalomyopathic form with methylmalonic aciduria
c2749864
449
gard
https://rarediseases.info.nih.gov/diseases/3681/mitochondrial-dna-depletion-syndrome-encephalomyopathic-form-with-methylmalonic-aciduria
2021-01-18T17:59:01
{"omim": ["245400"], "umls": ["C2749864"], "synonyms": ["Mitochondrial encephalomyopathy aminoacidopathy", "Mitochondrial dna depletion syndrome, encephalomyopathic form with methylmalonic aciduria, autosomal recessive", "Booth-Haworth-Dilling syndrome", "mtDNA depletion syndrome, encephalomyopathic form with methylmal...
Scalp–ear–nipple syndrome Other namesFinlay-Marks syndrome Scalp–ear–nipple syndrome is inherited in an autosomal dominant manner[1] Scalp–ear–nipple syndrome (also known as "Finlay–Marks syndrome") is a condition associated with aplasia cutis congenita.[2] ## Contents * 1 Presentation * 2 Genetics ...
Scalp–ear–nipple syndrome
c1867020
450
wikipedia
https://en.wikipedia.org/wiki/Scalp%E2%80%93ear%E2%80%93nipple_syndrome
2021-01-18T19:06:52
{"gard": ["159"], "mesh": ["C536623"], "umls": ["C1867020"], "orphanet": ["2036"], "wikidata": ["Q7429841"]}
A number sign (#) is used with this entry because of evidence that autosomal dominant mental retardation-56 (MRD56) is caused by heterozygous mutation in the CLTC gene (118955) on chromosome 17q23. Clinical Features DeMari et al. (2016) reported a 3.5-year-old girl, conceived through in vitro fertilization, wit...
MENTAL RETARDATION, AUTOSOMAL DOMINANT 56
c4693389
451
omim
https://www.omim.org/entry/617854
2019-09-22T15:44:38
{"doid": ["0080226"], "omim": ["617854"], "orphanet": ["178469", "442835"], "synonyms": ["Undetermined EOEE"]}
Typical hemolytic-uremic syndrome (typical HUS) is a thrombotic microangiopathy characterized by mechanical hemolytic anemia, thrombocytopenia, and renal dysfunction that is usually associated with prodromal enteritis caused by Shigella dysentriae type 1 or E. Coli. *[v]: View this template *[t]: Discuss this te...
Shiga toxin-associated hemolytic uremic syndrome
c1856143
452
orphanet
https://www.orpha.net/consor/cgi-bin/OC_Exp.php?lng=EN&Expert=90038
2021-01-23T18:54:12
{"omim": ["235400"], "icd-10": ["D58.8"], "synonyms": ["D+ HUS", "EHEC-HUS", "Hemolytic uremic syndrome associated with Shiga toxin-producing Escherichia coli", "Hemolytic uremic syndrome with diarrhea", "STEC-HUS", "Shiga-like toxin-associated HUS", "Stx-HUS", "Typical HUS", "Typical hemolytic uremic syndrome"]}
A number sign (#) is used with this entry because variation in the NPSR1 gene (608595) has been implicated in susceptibility to asthma-related traits linked to the 7p15-p14 region. Description Asthma-related traits include clinical symptoms of asthma, such as coughing, wheezing, and dyspnea; bronchial hyperresponsi...
ASTHMA-RELATED TRAITS, SUSCEPTIBILITY TO, 2
c1837811
453
omim
https://www.omim.org/entry/608584
2019-09-22T16:07:36
{"omim": ["608584"], "synonyms": ["Alternative titles", "ASRT2"]}
This article is about the contagious skin disease. For the change in state from liquid to gas, see Boiling. For other uses, see Boil (disambiguation). Boils Furuncle SpecialtyDermatology General surgery SymptomsPainful, small, rough skin growth Causesbacteria Ingrown hair A boil, also called a furuncle,...
Boil
c0242301
454
wikipedia
https://en.wikipedia.org/wiki/Boil
2021-01-18T18:37:04
{"mesh": ["D005667"], "icd-9": ["680.9"], "icd-10": ["L02"], "wikidata": ["Q193918"]}
A number sign (#) is used with this entry because of evidence that retinal dystrophy and iris coloboma with or without congenital cataract (RDICC) is caused by heterozygous mutation in the MIR204 gene (610942) on chromosome 9q21. One such family has been reported. Clinical Features Conte et al. (2015) reported ...
RETINAL DYSTROPHY AND IRIS COLOBOMA WITH OR WITHOUT CONGENITAL CATARACT
c4225233
455
omim
https://www.omim.org/entry/616722
2019-09-22T15:48:06
{"omim": ["616722"], "orphanet": ["488197"], "synonyms": []}
A rare, systemic amyloidosis characterized by slowly progressive renal disease presenting with proteinuria, hypertension and decreased glomerular filtration rate leading to progressive renal failure. Histology reveals amyloid deposits of leukocyte chemotactic factor-2 protein in the renal cortical interstitium, tubul...
ALECT2 amyloidosis
None
456
orphanet
https://www.orpha.net/consor/cgi-bin/OC_Exp.php?lng=EN&Expert=439224
2021-01-23T17:58:11
{"icd-10": ["E85.8"], "synonyms": ["Leukocyte chemotactic factor-2 amyloidosis"]}
This article needs more medical references for verification or relies too heavily on primary sources. Please review the contents of the article and add the appropriate references if you can. Unsourced or poorly sourced material may be challenged and removed. Find sources: "Pregnancy over age 50" – news · newspapers...
Pregnancy over age 50
None
457
wikipedia
https://en.wikipedia.org/wiki/Pregnancy_over_age_50
2021-01-18T18:34:22
{"wikidata": ["Q2372105"]}
This article needs more medical references for verification or relies too heavily on primary sources. Please review the contents of the article and add the appropriate references if you can. Unsourced or poorly sourced material may be challenged and removed. Find sources: "Throat irritation" – news · newspapers · b...
Throat irritation
c0700184
458
wikipedia
https://en.wikipedia.org/wiki/Throat_irritation
2021-01-18T18:28:51
{"umls": ["C0700184"], "wikidata": ["Q7798315"]}
Uremic pericarditis Pericarditis fibrinosa SpecialtyUrology Uremic pericarditis is a form of pericarditis. It causes fibrinous pericarditis.[1] The main cause of the disease is poorly understood.[2] ## Contents * 1 Signs and symptoms * 2 Pathology * 3 Treatment * 4 References * 5 External links ...
Uremic pericarditis
c0265152
459
wikipedia
https://en.wikipedia.org/wiki/Uremic_pericarditis
2021-01-18T19:03:55
{"icd-10": ["N18.8"], "wikidata": ["Q7900393"]}
Beta-propeller protein-associated neurodegeneration (BPAN), also known as static encephalopathy of childhood with neurodegeneration in adulthood, is a rare form of neurodegeneration with brain iron accumulation (NBIA) characterized by early-onset developmental delay and further neurological deterioration in early...
Beta-propeller protein-associated neurodegeneration
c3550973
460
orphanet
https://www.orpha.net/consor/cgi-bin/OC_Exp.php?lng=EN&Expert=329284
2021-01-23T18:56:58
{"gard": ["12570"], "omim": ["300894"], "icd-10": ["G23.0"], "synonyms": ["BPAN", "NBIA5", "Neurodegeneration with brain iron accumulation type 5", "SENDA", "Static encephalopathy of childhood with neurodegeneration in adulthood"]}
Ocular albinism SpecialtyEndocrinology Ocular albinism is a form of albinism which, in contrast to oculocutaneous albinism, presents primarily in the eyes.[1] There are multiple forms of ocular albinism, which are clinically similar.[2]:865 Both known genes are on the X chromosome. When the term "autosomal re...
Ocular albinism
c0078917
461
wikipedia
https://en.wikipedia.org/wiki/Ocular_albinism
2021-01-18T19:04:14
{"mesh": ["D016117"], "icd-9": ["270.2"], "icd-10": ["E70.3"], "orphanet": ["284804"], "wikidata": ["Q2831905"]}
Yunis et al. (1980) described a Colombian family in which 10 males in 3 generations, in a typical X-linked recessive pedigree pattern, had the Dyggve-Melchior-Clausen syndrome. The affected males varied in age from 13 to 15 years. Normal intelligence was another difference from the autosomal recessive form. The a...
DYGGVE-MELCHIOR-CLAUSEN SYNDROME, X-LINKED
c0265286
462
omim
https://www.omim.org/entry/304950
2019-09-22T16:18:20
{"mesh": ["C535726"], "omim": ["304950"], "orphanet": ["239"]}
A number sign (#) is used with this entry because of evidence that hypotonia, hypoventilation, impaired intellectual development, dysautonomia, epilepsy, and eye abnormalities (HIDEA) is caused by homozygous or compound heterozygous mutation in the P4HTM gene (614584) on chromosome 3p21. Description Hypotonia, hypo...
HYPOTONIA, HYPOVENTILATION, IMPAIRED INTELLECTUAL DEVELOPMENT, DYSAUTONOMIA, EPILEPSY, AND EYE ABNORMALITIES
None
463
omim
https://www.omim.org/entry/618493
2019-09-22T15:41:40
{"omim": ["618493"]}
## Summary ### Clinical characteristics. Ataxia with oculomotor apraxia type 1 (AOA1) is characterized by childhood onset of slowly progressive cerebellar ataxia, followed by oculomotor apraxia and a severe primary motor peripheral axonal motor neuropathy. The first manifestation is progressive gait imbalance (mean...
Ataxia with Oculomotor Apraxia Type 1
c1859598
464
gene_reviews
https://www.ncbi.nlm.nih.gov/books/NBK1456/
2021-01-18T21:42:33
{"mesh": ["C538013"], "synonyms": ["AOA1"]}
Small patella syndrome (SPS) is a rare syndrome that mainly affects the way certain bones are formed (developed). A person with SPS usually has very small kneecaps (hypoplastic patella) or may have no kneecaps at all (aplastic). The hip (pelvic) bones may be weak, and the bones of the feet may not have formed correct...
Small patella syndrome
c1840061
465
gard
https://rarediseases.info.nih.gov/diseases/3030/small-patella-syndrome
2021-01-18T17:57:40
{"mesh": ["C535540"], "omim": ["147891"], "umls": ["C1840061"], "orphanet": ["1509"], "synonyms": ["Scott-Taor syndrome", "Coxo-podo-patellar syndrome", "Ischiopatellar dysplasia", "Patella aplasia, coxa vara, tarsal synostosis", "Congenital coxa vara, patella aplasia and tarsal synostosis", "ischiocoxopodopatellar syn...
A rare, malignant splenic B-cell lymphoma/leukemia characterized by circulating abnormal lymphocytes with intermediate morphology between prolymphocytes and hairy cells with positive expression of CD11c and negative expression of CD25, CD123 and the BRAFV600E mutation. Manifestations include splenomegaly, elevated wh...
Hairy cell leukemia variant
c0349633
466
orphanet
https://www.orpha.net/consor/cgi-bin/OC_Exp.php?lng=EN&Expert=300878
2021-01-23T18:35:17
{"umls": ["C0349633"], "icd-10": ["C91.4"], "synonyms": ["HCL-v", "Leukemic reticuloendotheliosis variant", "Prolymphocytic variant of HCL", "Prolymphocytic variant of hairy cell leukemia"]}
This article is about the wrist fracture. For the vertebral fracture, see Smith fracture. Smith's fracture Other namesReverse Colles' fracture, Goyrand-Smith's Smith's fracture SpecialtyOrthopedics A Smith's fracture, is a fracture of the distal radius.[1] It is caused by a direct blow to the dorsal forea...
Smith's fracture
c0347795
467
wikipedia
https://en.wikipedia.org/wiki/Smith%27s_fracture
2021-01-18T18:35:09
{"umls": ["C0347795"], "icd-9": ["813.41"], "icd-10": ["S52.5"], "wikidata": ["Q3752481"]}
Atypical Rett syndrome is a neurodevelopmental disorder that is diagnosed when a child has some of the symptoms of Rett syndrome but does not meet all the diagnostic criteria. Like the classic form of Rett syndrome, atypical Rett syndrome mostly affects girls. Children with atypical Rett syndrome can have symptom...
Atypical Rett syndrome
c2748910
468
gard
https://rarediseases.info.nih.gov/diseases/4694/atypical-rett-syndrome
2021-01-18T18:01:59
{"mesh": ["C567576"], "umls": ["C2748910"], "orphanet": ["3095"], "synonyms": ["Rett like syndrome", "Rett syndrome variant"]}
Apraxia Apraxia is characterized by loss of the ability to execute or carry out learned purposeful movements SpecialtyNeurology, Psychiatry TreatmentPhysical therapy Apraxia is a motor disorder caused by damage to the brain (specifically the posterior parietal cortex or corpus callosum[1]) in which the ind...
Apraxia
c3495144
469
wikipedia
https://en.wikipedia.org/wiki/Apraxia
2021-01-18T18:40:53
{"gard": ["5838"], "mesh": ["D001072"], "icd-9": ["784.69", "438.81"], "icd-10": ["R48.2"], "wikidata": ["Q498916"]}
## Clinical Features Teebi et al. (1988) described an Arabic family with normal consanguineous parents and 5 children (3 males and 2 females) with some features of Aarskog syndrome (see 305400) in addition to unusual hair changes. The hair was coarse, dry, and relatively hypopigmented with widow's peak trait (1...
FACIODIGITOGENITAL SYNDROME, AUTOSOMAL RECESSIVE
c1856871
470
omim
https://www.omim.org/entry/227330
2019-09-22T16:27:58
{"mesh": ["C535331"], "omim": ["227330"], "orphanet": ["1974"], "synonyms": ["Alternative titles", "AARSKOG-LIKE SYNDROME", "KUWAIT TYPE FACIODIGITOGENITAL SYNDROME"]}
A rare neoplastic disease characterized by the presence of ten or more hepatocellular adenomas in a background of normal appearing hepatic parenchyma. The majority of reported cases are female. There is no association with steroid use. The condition is considered benign, although the risk of complications (such as ma...
Liver adenomatosis
None
471
orphanet
https://www.orpha.net/consor/cgi-bin/OC_Exp.php?lng=EN&Expert=566841
2021-01-23T18:14:09
{"synonyms": ["Hepatic adenomatosis"]}
Whitehouse et al. (1988) studied the family of the fourth human example of anti-LKE (originally called Luke) and excluded close linkage to MNS, Rh, HLA, PI, Gm, and C6. They also showed that LKE is genetically independent of P1, K, Xg, Au, Se, and C3. *[v]: View this template *[t]: Discuss this template *[...
BLOOD GROUP--LKE
c1862216
472
omim
https://www.omim.org/entry/111130
2019-09-22T16:44:20
{"omim": ["111130"], "synonyms": ["Alternative titles", "BLOOD GROUP--LUKE, FORMERLY"]}
Prevalence of AIDS in Nigeria from 1991–2010. Includes predictions up to 2018. HIV/AIDS in Nigeria was a concern in the 2000s, when an estimated seven million people had HIV/AIDS. In 2008, the HIV prevalence rate among adults aged between 15 and 49 was 3.9 percent, in 2018 the rate among adults aged between 15 and 6...
HIV/AIDS in Nigeria
None
473
wikipedia
https://en.wikipedia.org/wiki/HIV/AIDS_in_Nigeria
2021-01-18T18:59:53
{"wikidata": ["Q5629871"]}
Charcot-Marie-Tooth disease, type 2B2 (CMT2B2, also referred to as CMT4C3) is an axonal CMT peripheral sensorimotor polyneuropathy that has been described in a large consanguineous Costa Rican family of Spanish ancestry. ## Clinical description Onset occurs in adulthood (between 26 and 42 years of age) with sym...
Charcot-Marie-Tooth disease type 2B2
c1854150
474
orphanet
https://www.orpha.net/consor/cgi-bin/OC_Exp.php?lng=EN&Expert=101101
2021-01-23T18:12:13
{"gard": ["1249"], "mesh": ["C537991"], "omim": ["605589"], "umls": ["C1854150"], "icd-10": ["G60.0"], "synonyms": ["AR-CMT2B2", "Autosomal recessive axonal CMT4C3", "Autosomal recessive axonal Charcot-Marie-Tooth disease type 2B2"]}
Waldmann disease Other namesWaldmann's disease, Primary intestinal lymphangiectasia Waldmann disease is a rare disease[1] characterized by enlargement of the lymph vessels supplying the lamina propria of the small intestine.[2] Although its prevalence is unknown, it being classified as a "rare disease" means ...
Waldmann disease
c2931241
475
wikipedia
https://en.wikipedia.org/wiki/Waldmann_disease
2021-01-18T19:10:43
{"gard": ["7873"], "mesh": ["C536567"], "umls": ["C2931241", "C0267372"], "icd-9": ["457.1"], "icd-10": ["I89.0"], "orphanet": ["90362"], "wikidata": ["Q7961531"]}
Inverse Marcus-Gunn phenomenon is a rare congenital synkinesis where jaw opening by the pterygoid muscle (during eating or yawning) causes eyelid drooping from inhibition of the oculomotor nerve to the levator palpebrae superioris. Familial occurrence has been reported. *[v]: View this template *[t]: Discuss...
Inverse Marcus-Gunn phenomenon
None
476
orphanet
https://www.orpha.net/consor/cgi-bin/OC_Exp.php?lng=EN&Expert=98951
2021-01-23T17:31:16
{"icd-10": ["Q07.8"]}
Distal myopathy refers to a group of muscle diseases which share the clinical pattern of predominant weakness and atrophy beginning in the feet and/or hands. ## Epidemiology The estimated prevalence of distal myopathy in the northern region of England is 1/300,000. Some forms have been identified with greater frequ...
Distal myopathy
c0751336
477
orphanet
https://www.orpha.net/consor/cgi-bin/OC_Exp.php?lng=EN&Expert=599
2021-01-23T18:09:32
{"mesh": ["D049310"], "umls": ["C0751336"], "icd-10": ["G71.0"], "synonyms": ["Distal muscular dystrophy"]}
3q29 microdeletion syndrome is a rare chromosome disorder. Symptoms may include delay reaching some developmental milestones such as sitting, walking or talking, frequent ear and respiratory infections, and a small head size (microcephaly). Some babies with this condition are born with a cleft lip or cleft palate, an...
3q29 microdeletion syndrome
c2674949
478
gard
https://rarediseases.info.nih.gov/diseases/11974/3q29-microdeletion-syndrome
2021-01-18T17:57:00
{"mesh": ["C567184"], "omim": ["609425"], "orphanet": ["65286"], "synonyms": ["3qter deletion", "Del(3)(q29)", "Monosomy 3qter", "Chromosome 3q29 microdeletion syndrome", "3q subtelomere deletion syndrome", "3q29 deletion", "Monosomy 3q29", "3q29 deletion syndrome"]}
Cochleosaccular degeneration-cataract syndrome is characterised by progressive sensorineural hearing loss due to severe cochleosaccular degeneration and cataract. So far, it has been reported in two families. Transmission is autosomal dominant. *[v]: View this template *[t]: Discuss this template *[e]: Edit th...
Cochleosaccular degeneration-cataract syndrome
c1861512
479
orphanet
https://www.orpha.net/consor/cgi-bin/OC_Exp.php?lng=EN&Expert=3233
2021-01-23T17:23:27
{"gard": ["9418"], "mesh": ["C536432"], "omim": ["120040"]}
A number sign (#) is used with this entry because of evidence that Cohen-Gibson syndrome (COGIS) is caused by heterozygous mutation in the EED gene (605984) on chromosome 11q14. Description Cohen-Gibson syndrome is an overgrowth disorder characterized by increased somatic parameters apparent from birth and associat...
COHEN-GIBSON SYNDROME
c4479654
480
omim
https://www.omim.org/entry/617561
2019-09-22T15:45:35
{"omim": ["617561"], "genereviews": ["NBK540017"]}
## Summary ### Clinical characteristics. Kindler syndrome (KS), a rare subtype of inherited epidermolysis bullosa, is characterized by skin fragility and acral blister formation beginning at birth, diffuse cutaneous atrophy, photosensitivity (which is most prominent during childhood and usually decreases after adol...
Kindler Syndrome
c0406557
481
gene_reviews
https://www.ncbi.nlm.nih.gov/books/NBK349072/
2021-01-18T21:15:47
{"mesh": ["C536321"], "synonyms": ["Congenital Bullous Poikiloderma"]}
A number sign (#) is used with this entry because of evidence that spastic paraplegia-6 (SPG6) is caused by heterozygous mutation in the NIPA1 gene (608145) on chromosome 15q11. For a general phenotypic description and a discussion of genetic heterogeneity of autosomal dominant spastic paraplegia, see SPG3A (182600)...
SPASTIC PARAPLEGIA 6, AUTOSOMAL DOMINANT
c1838192
482
omim
https://www.omim.org/entry/600363
2019-09-22T16:16:13
{"doid": ["0110811"], "mesh": ["C536866"], "omim": ["600363"], "orphanet": ["100988"], "synonyms": ["Alternative titles", "FAMILIAL SPASTIC PARAPLEGIA, AUTOSOMAL DOMINANT, 3"]}
A number sign (#) is used with this entry because of evidence that an intellectual developmental disorder with autism and speech delay (IDDAS) is caused by heterozygous mutation in the TBR1 gene (604616) on chromosome 2q. Description IDDAS is a neurodevelopmental disorder characterized by varying degrees of intelle...
INTELLECTUAL DEVELOPMENTAL DISORDER WITH AUTISM AND SPEECH DELAY
c1853755
483
omim
https://www.omim.org/entry/606053
2019-09-22T16:10:47
{"omim": ["606053"], "synonyms": ["Alternative titles", "PHRASE SPEECH DELAY, AUTISM-RELATED", "AUTISM-RELATED SPEECH DELAY", "AUTISM, SUSCEPTIBILITY TO, 5, FORMERLY"]}
In 3 brothers and a sister out of 9 sibs of nonconsanguineous parents, Richieri-Costa et al. (1984) described a progressive disorder with onset usually at a mean age of 5.7 years and including myotonia, progressive impairment of gait, alterations in the thorax and vertebral column, short stature, and mild to moderate...
MYOTONIA WITH SKELETAL ABNORMALITIES AND MENTAL RETARDATION
c2930978
484
omim
https://www.omim.org/entry/255710
2019-09-22T16:24:34
{"mesh": ["C535675"], "omim": ["255710"], "orphanet": ["3101"]}
Norrie disease SpecialtyMedical genetics Norrie disease is a rare disease and genetic disorder that primarily affects the eyes and almost always leads to blindness. It is caused by mutations in the Norrin cystine knot growth factor (NDP) gene, which is located on the X chromosome.[1][2] In addition to the cong...
Norrie disease
c0266526
485
wikipedia
https://en.wikipedia.org/wiki/Norrie_disease
2021-01-18T18:44:32
{"gard": ["7224"], "mesh": ["C537849"], "umls": ["C0266526"], "icd-9": ["743.8"], "orphanet": ["649"], "wikidata": ["Q1415842"]}
Caudal regression-sirenomelia spectrum is a group of rare genetic developmental defect during embryogenesis disorders characterized by varying degrees of caudal abdomen, pelvic, renal, anorectal, urogenital and/or lumbosacral spine malformations, with or without lower limb fusion. Phenotype is highly variable ran...
Caudal regression-sirenomelia spectrum
None
486
orphanet
https://www.orpha.net/consor/cgi-bin/OC_Exp.php?lng=EN&Expert=444941
2021-01-23T18:43:24
{}
A number sign (#) is used with this entry because of evidence that multiple congenital anomalies-hypotonia-seizures syndrome-3 (MCAHS3) is caused by homozygous or compound heterozygous mutation in the PIGT gene (610272) on chromosome 20q13. Description Multiple congenital anomalies-hypotonia-seizures syndrome is an...
MULTIPLE CONGENITAL ANOMALIES-HYPOTONIA-SEIZURES SYNDROME 3
c3809356
487
omim
https://www.omim.org/entry/615398
2019-09-22T15:52:26
{"doid": ["0080140"], "omim": ["615398"], "orphanet": ["369837"], "synonyms": ["Congenital disorder of glycosylation due to PIGT deficiency", "Multiple congenital anomalies-hypotonia-seizures syndrome type 3", "PIGT-CDG", "MCAHS type 3", "Alternative titles", "GLYCOSYLPHOSPHATIDYLINOSITOL BIOSYNTHESIS DEFECT 7"]}
## Summary ### Clinical characteristics. Autosomal dominant tubulointerstitial kidney disease, MUC1-related (ADTKD-MUC1) was previously known as medullary cystic kidney disease type 1. It is characterized by slowly progressive tubulointerstitial disease that leads to end-stage renal disease (ESRD) and the need for ...
Autosomal Dominant Tubulointerstitial Kidney Disease, MUC1-Related
c1868139
488
gene_reviews
https://www.ncbi.nlm.nih.gov/books/NBK153723/
2021-01-18T21:41:25
{"mesh": ["C536137"], "synonyms": ["ADTKD-MUC1", "MCKD1", "Medullary Cystic Kidney Disease Type 1"]}
Beare–Stevenson cutis gyrata syndrome Other namesCutis gyrata-acanthosis nigricans-craniosynostosis syndrome[1] Diagnostic methodidentification of the p.Pro250Arg pathogenic variant in FGFR3; the diagnosis of FGFR2-related isolated coronal synostosis is based on identification of a FGFR2 pathogenic variant. The d...
Beare–Stevenson cutis gyrata syndrome
c1852406
489
wikipedia
https://en.wikipedia.org/wiki/Beare%E2%80%93Stevenson_cutis_gyrata_syndrome
2021-01-18T18:38:37
{"gard": ["332"], "mesh": ["C565129"], "umls": ["C1852406"], "orphanet": ["1555"], "wikidata": ["Q4876717"]}
Tracheobronchomalacia Other namesExcessive Dynamic Airway Collapse Trachea anatomy SpecialtyPulmonology SymptomsChronic cough, stridor, inability to raise secretions, breathlessness Usual onsetFrom birth (Congenital ), Adulthood (Acquired) DurationCongenital: Significant improvement after 18-24 months...
Tracheobronchomalacia
c0340231
490
wikipedia
https://en.wikipedia.org/wiki/Tracheobronchomalacia
2021-01-18T18:55:45
{"gard": ["7791"], "mesh": ["D055089"], "umls": ["C0340231"], "wikidata": ["Q7831316"]}
A form of congenital disorders of N-linked glycosylation characterized by facial dysmorphism (prominent forehead, large ears, thin upper lip), generalized hypotonia, feeding difficulties, moderate to severe developmental delay, progressive microcephaly, frequent upper respiratory tract infections due to impaired ...
ALG12-CDG
c2931001
491
orphanet
https://www.orpha.net/consor/cgi-bin/OC_Exp.php?lng=EN&Expert=79324
2021-01-23T18:53:08
{"gard": ["9833"], "mesh": ["C535745"], "omim": ["607143"], "umls": ["C2931001"], "icd-10": ["E77.8"], "synonyms": ["CDG syndrome type Ig", "CDG-Ig", "CDG1G", "Carbohydrate deficient glycoprotein syndrome type Ig", "Congenital disorder of glycosylation type 1g", "Congenital disorder of glycosylation type Ig", "Mannosyl...
Progressive microcephaly-seizures-cortical blindness-developmental delay syndrome is a rare, genetic, neuro-ophthalmological syndrome characterized by post-natal, progressive microcephaly and early-onset seizures, associated with delayed global development, bilateral cortical visual impairment and moderate to severe ...
Progressive microcephaly-seizures-cortical blindness-developmental delay syndrome
c4225261
492
orphanet
https://www.orpha.net/consor/cgi-bin/OC_Exp.php?lng=EN&Expert=477814
2021-01-23T16:55:54
{"omim": ["616632"]}
Epithelioid cell histiocytoma SpecialtyDermatology Epithelioid cell histiocytoma is a rare skin condition that is considered to be a variant of a dermatofibroma.[1] ## See also[edit] * Pleomorphic lipoma * List of cutaneous conditions ## References[edit] 1. ^ Rapini, Ronald P.; Bolognia, Jean L.; Jor...
Epithelioid cell histiocytoma
c1275242
493
wikipedia
https://en.wikipedia.org/wiki/Epithelioid_cell_histiocytoma
2021-01-18T18:54:21
{"umls": ["C1275242"], "wikidata": ["Q5383706"]}
Oculotrichoanal syndrome is a form of rare, multiple congenital anomalies/dysmorphic syndrome characterized by a combination of various nose, eye, gastrointestinal and genitourinary abnormalities. Clinical presentation is variable and often includes bifid and broad nasal tip, aberrant anterior hairline, coloboma,...
Oculotrichoanal syndrome
c1855425
494
orphanet
https://www.orpha.net/consor/cgi-bin/OC_Exp.php?lng=EN&Expert=2717
2021-01-23T18:22:13
{"gard": ["3395"], "mesh": ["C536022"], "omim": ["248450"], "umls": ["C1855425"], "icd-10": ["Q87.8"], "synonyms": ["MOTA syndrome", "Manitoba oculotrichoanal syndrome", "Marles syndrome", "Marles-Greenberg-Persaud syndrome"]}
A rare, genetic, constitutional thrombocytopenia disease characterized by mild to moderate thrombocytopenia, abnormal platelet function and a propensity to develop hematological malignancies, mainly of myeloid origin. ## Epidemiology The estimated prevalence at birth is less than 1/ 1 000 000. There are no availabl...
Familial platelet disorder with associated myeloid malignancy
c1832388
495
orphanet
https://www.orpha.net/consor/cgi-bin/OC_Exp.php?lng=EN&Expert=71290
2021-01-23T18:45:14
{"gard": ["10352"], "mesh": ["C563324"], "omim": ["601399", "616216"], "umls": ["C1832388"], "icd-10": ["D69.4"], "synonyms": ["FPD/AML", "FPDMM", "FPS/AML", "Familial platelet disorder with predisposition to acute myelogenous leukemia", "Familial platelet disorder with predisposition to myeloid malignancy", "Familial ...
Amyoplasia is a condition characterized by a generalized lack in the newborn of muscular development and growth, with contracture and deformity at most joints. It is the most common form of arthrogryposis.[1] It is characterized by the four limbs being involved, and by the replacement of skeletal muscle by dense fib...
Amyoplasia
c0432185
496
wikipedia
https://en.wikipedia.org/wiki/Amyoplasia
2021-01-18T19:05:22
{"orphanet": ["488586"], "synonyms": ["Amyoplasia congenita"], "wikidata": ["Q4749575"]}
A rare congenital malformation where a second structure with individual lumen and stratified squamous mucosa and muscularis mucosa lies within or adjacent to the true esophagus causing dysphagia, nausea, vomiting, retrosternal pain and respiratory problems (stridor and recurrent pneumonia) and usually presenting in c...
Tubular duplication of the esophagus
None
497
orphanet
https://www.orpha.net/consor/cgi-bin/OC_Exp.php?lng=EN&Expert=100048
2021-01-23T17:15:12
{"icd-10": ["Q39.8"]}
Dermatofibroma Other namesDermal dendrocytoma,[1] Dermatofibroma,[2] Fibrous dermatofibroma,[2] Fibrous histiocytoma,[2] Fibroma simplex,[1] Nodular subepidermal fibrosis,[1] and Sclerosing hemangioma[1]) SpecialtyOncology A dermatofibroma, or benign fibrous histiocytomas, is a benign skin growth.[3] ## Con...
Dermatofibroma
c1509148
498
wikipedia
https://en.wikipedia.org/wiki/Dermatofibroma
2021-01-18T18:49:59
{"umls": ["C1509148"], "icd-9": ["216.9"], "icd-10": ["D23"], "wikidata": ["Q1200237"]}
In the legal profession and courts, a querulant (from the Latin querulus \- "complaining") is a person who obsessively feels wronged, particularly about minor causes of action. In particular the term is used for those who repeatedly petition authorities or pursue legal actions based on manifestly unfounded ground...
Querulant
None
499
wikipedia
https://en.wikipedia.org/wiki/Querulant
2021-01-18T18:57:34
{"wikidata": ["Q252110"]}