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Entry Name Description Category Pathway
H00443 Osteoglophonic dysplasia
Osteoglophonic dwarfism
Osteoglophonic dysplasia (OD) or osteoglophonic dwarfism (OGD) is an autosomal dominant disorder that has skeletal phenotypes of craniosynostosis. Missense mutation of FGFR1 has been reported. Congenital malformation
H00444 Osteopathia striata with cranial sclerosis Osteopathia striata with cranial sclerosis (OSCS) is a bone dysplasia characterized by longitudinal striations in the metaphyseal region of the long bones and sclerosis of the craniofacial bones. The condition ... Congenital malformation
H00445 Osteoarthritis with mild chondrodysplasia Osteoarthritis with mild chondrodysplasia (OSCDP) is characterized by a progressive degeneration of the articular cartilages of joints with mild spinal chondrodysplasia due to the mutation of type II procollagen ... Congenital malformation
H00446 Craniofacial-deafness-hand syndrome Craniofacial-deafness-hand syndrome is inherited as an autosomal dominant or X-linked mutation characterized by a flat facial profile, hypoplastic nose, and a sensorineural hearing loss. A missense mutation ... Congenital malformation
H00447 HEM skeletal dysplasia
Greenberg dysplasia
Hydrops ectopic calcification-moth-eaten (HEM) or Greenberg skeletal dysplasia is a lethal chondrodystrophy characterized by fetal hydrops, short limbs, and abnormal chondro-osseous calcification. It is ... Congenital malformation
H00448 Familial osteochondritis dissecans
Osteochondritis dissecans, short stature, and early-onset osteoarthritis
Osteochondritis dissecans is defined as a separation of articular cartilage and subchondral bone from the joint surface, affecting the knee, ankle and elbow joints. The disease is caused by heterozygous ... Musculoskeletal disease
H00449 Oculodentodigital dysplasia Oculodentodigital dysplasia (ODDD) is an inherited disorder involving characteristic facial appearance and abnormalities of eyes, teeth, and limbs. The disease is inherited in both an autosomal dominant ... Congenital malformation
H00450 Worth type autosomal dominant osteosclerosis
Endosteal hyperostosis
Worth type autosomal dominant osteosclerosis, also known as endosteal hyperostosis, is a genetic disorder characterized by high bone density. Craniofacial anomalies develop during adolescence. This disease ... Congenital malformation
H00451 Osteoporosis-pseudoglioma syndrome Osteoporosis-pseudoglioma syndrome (OPPG) is inherited as an autosomal recessive condition and is characterized by severe congenital osteoporosis with blindness. Mutations in LRP5 cause OPPG. Congenital malformation
H00452 Buschke-Ollendorff syndrome
Osteopoikilosis
Buschke-Ollendorff syndrome (BOS), also known as Osteopoikilosis, is a disorders characterized by increased bone density. Loss-of-function mutations in LEMD3, which encodes an inner nuclear membrane protein ... Congenital malformation
H00453 Branchio-oto-renal syndrome
BOR syndrome
Branchio-oto-renal (BOR) syndrome and Branchiootic (BO) syndrome show overlapping phenotypes of bilateral conductive hearing loss, branchial defects, and facial abnormalities. Individuals with BOR syndrome ... Congenital malformation
H00454 Oral-facial-digital syndrome
Orofaciodigital syndrome
Oral-facial-digital syndrome is a group of heterogeneous disorders characterized by malformations of the face, oral cavity and digits. OFD type I is a male lethal disorder and due to mutations in the OFD1 ... Congenital malformation
H00455 Spinal muscular atrophy Spinal muscular atrophy (SMA) is a neuromuscular disease characterized by degeneration of motor neurons, resulting in progressive muscle atrophy and paralysis. The most common form of SMA is caused by ... Neurodegenerative disease
H00456 Fronto-otopalatodigital syndromes Fronto-Otopalatodigital Osteodysplasia comprises four disorders that arise from missense mutations in FLNA encoding the actin-binding cytoskeletal protein filamin A. The disorders are inherited in X-linked ... Congenital malformation
H00457 Primary hypertrophic osteoarthropathy Primary hypertrophic osteoarthropathy (PHO) is a familial disorder with delayed cranial suture closure, digital clubbing, arthropathy, acro-osteolysis, periostosis, and pachydermia. Mutations in HPGD gene ... Musculoskeletal disease
H00458 Syndromic craniosynostoses Craniosynostosis is the premature fusion of the cranial sutures and secondary distortion of skull shape. Syndromic craniosynostosis typically involves cranial sutures plus central nervous system and extracranial ... Congenital malformation
H00459 Synpolydactyly Synpolydactyly (SPD) is a dominantly inherited congenital limb malformation showing digit duplication and webbing of third and fourth fingers. Mutation in HOXD13 induces synpolydactyly. Synpolydactyly ... Congenital malformation
H00460 Hand-foot-genital syndrome Hand-foot-genital syndrome is very rare dominantly inherited condition affecting the development of the limbs and genitourinary tract. Congenital malformation
H00461 Ischiocoxopodopatellar syndrome
Coxopodopatellar syndrome
Small patella syndrome
Scott-Taor syndrome
Ischiocoxopodopatellar syndrome, also known as small patella syndrome, is a skeletal dysplasia with anomalies of the pelvis. Ossification of the ischia and inferior pubic rami is also disrupted in patients Congenital malformation
H00462 Stuve-Wiedemann syndrome Stuve-Wiedemann syndrome is an autosomal recessively inherited disorder characterized by congenital bone dysplasia like bowing of the long bones. Mutations in LIFR result in this disease. Congenital malformation
H00463 Currarino syndrome Currarino syndrome is a condition characterized by the combination of sacral malformation, hindgut anomaly, and presacral mass. The HLXB9 gene is responsible for the syndrome. Congenital malformation
H00464 Nail-patella syndrome Nail-patella syndrome is a condition caused by mutation in LMX1B that regulates COL4A4 and COL4A3 expression. The most common associated abnormality is nail dysplasia. Congenital malformation
H00465 Fragile X syndrome Fragile X syndrome (FXS) is a cognitive disorder caused by silencing of the fragile X mental retardation 1 gene (FMR1). Absence of the associated protein FMRP leads to the dysregulation of many genes creating ... Chromosomal abnormality
H00466 Grebe dysplasia
Acromesomelic dysplasia Hunter-Thompson type
Acromesomelic dysplasia is a rare form of severe acromesomelic limb shortening inherited in autosomal recessive fashion. Lower limbs are more affected than the upper limbs, showing nonfunctional fingers ... Congenital malformation
H00467 Fibular hypoplasia and complex brachydactyly
Du Pan syndrome
Acromesomelic dysplasia 2B
Fibular hypoplasia and complex brachydactyly (DuPan syndrome), inherited as an autosomal recessive trait, is characterized by fibula aplasia and severe limb shortening. Affected individuals were reported ... Congenital malformation
H00468 Acromesomelic dysplasia, Demirhan type
Acromesomelic dysplasia with genital anomalies
Acromesomelic dysplasia with genital anomalies is a condition that shows severe limb malformation with brachydactyly. Hypogonadism is also present. It is caused by homozygous mutation of BMPR1B. Congenital malformation
H00469 Mitochondrial DNA depletion syndrome Mitochondrial DNA depletion syndromes (MDSs) are a group of heterogeneous autosomal recessive disorders associated with a severe reduction in mitochondrial DNA in the affected tissues. The manifestations ... Inherited metabolic disorder, Mitochondrial disease
H00470 Acromesomelic dysplasia, Maroteaux type Acromesomelic dysplasia, Maroteaux type is an autosomal recessive skeletal dysplasia that affects postnatal skeletal growth. Affected individuals show marked short stature and limb shortening. Homozygous ... Congenital malformation
H00471 Split-hand/foot malformation Split hand/foot malformation (SHFM) is a congenital limb malformation characterized by median clefts of hands and foot. Disrupted formation of the apical ectodermal ridge during development results in ... Congenital malformation
H00472 Torg syndrome
Torg-Winchester syndrome
Multicentric osteolysis, nodulosis, and arthropathy
Torg syndrome, also known as Multicentric osteolysis, nodulosis, and arthropathy (MONA) is a multicentric osteolysis syndrome characterized by progressive bone loss in hands and feet. MMP2 mutations are ... Musculoskeletal disease
H00473 Mitochondrial complex I deficiency Mitochondrial complex I deficiency, the most common mitochondrial disorders, is a group of highly heterogeneous conditions characterised by faulty oxidative phosphorylation (OXPHOS). Human complex I is ... Inherited metabolic disorder, Mitochondrial disease
H00474 Schneckenbecken dysplasia Schneckenbecken dysplasia is a severe skeletal dysplasia that leads to perinatal death. The characteristic is the snail-like configuration of the hypoplastic iliac bones. Patients also have limbs with ... Congenital malformation
H00475 Enlarged parietal foramina/cranium bifidum Enlarged parietal foramina/cranium bifidum is characterised by oval defects in the parietal bones. It results from incomplete ossification of the parietal bones during development and is caused by ALX4 ... Congenital malformation
H00476 Multiple epiphyseal dysplasia Multiple epiphyseal dysplasia (EDM) is a genetically heterogeneous condition where ossification of epiphyses is delayed. Mutations causing EDM have been identified in COMP, DTDST, MATN3, COL9A1, COL9A2 ... Congenital malformation
H00477 Pseudoachondroplasia Pseudoachondroplasia (PSACH) is a condition with short-limb, short stature, joint pain, and early-onset osteoarthrosis caused by epiphyseal ossification delay. PSACH is caused by mutations in COMP. Congenital malformation
H00478 Prader-Willi syndrome Prader-Willi syndrome (PWS) and Angelman syndrome (AS) are the most studied genomic-imprinting disorders mapped to chromosome 15q11-q13. Lack of a functional paternal copy of 15q11-q13 causes PWS. Additionally ... Chromosomal abnormality
H00479 Metaphyseal dysplasias Metaphyseal dysplasias are very rare skeletal disorders with short limb/short stature phenotypes. Defects in metaphyseal development leads to enlarged metaphyses of long bones that tend to fracture in ... Congenital malformation
H00480 X-linked intellectual developmental disorder
X-linked mental retardation
X-linked intellectual developmental disorder (XLID), formerly known as X-linked mental retardation, is an inherited condition that causes failure to develop cognitive abilities because of mutations in ... Mental and behavioural disorder
H00481 Cone-rod dystrophy and cone dystrophy Cone-rod dystrophy (CORD) and cone dystrophy (COD) are a subgroup of inherited retinal dystrophies characterized by progressive loss of photoreceptor function. In contrast to retinitis pigmentosa (RP) ... Nervous system disease
H00482 Brachydactyly Brachydactyly (BD) comprises hereditary limb malformations characterized by apparent shortening of digits. Bone dysostosis is seen in middle phalanges in type A; distal phalanges in type B; distal phalanx ... Congenital malformation
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