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Entry Name Description Category Pathway
H01204 Cerebellar ataxia, mental retardation (MR), and dysequilibrium syndrome (CAMRQ) Cerebellar ataxia, mental retardation (MR), and dysequilibrium syndrome (CAMRQ) is autosomal recessive, genetically heterogeneous conditions characterized by early onset of cerebellar ataxia and MR. Patients ... Nervous system disease
H01205 Coumarin resistance
Warfarin resistance
Warfarin is widely prescribed anticoagulant for the prevention of thromboembolic diseases. However, its use is made difficult by the wide interindividual variation in dose required to achieve a therapeutic ... Inherited metabolic disorder
H01206 Plasminogen deficiency Plasminogen deficiency is characterized by decreased serum plasminogen activity. Two forms of the disorder are distinguished. Type 1, also called hypoplasminogenemia, is associated with pseudomembrane ... Hematologic disease
H01207 Trigonocephaly Trigonocephaly is a rare form of craniosynostosis characterized by the premature closure of the metopic suture. It has been reported that mutations in FGFR1 and FREM1 can be associated with trigonocephaly Congenital malformation
H01208 Globozoospermia
Round-headed spermatozoa
Globozoospermia is a rare form of teratozoospermia, mainly characterized by round-headed spermatozoa that lack an acrosome. Acrosome plays an important role at the site of sperm-zona pellucida binding ... Reproductive system disease
H01209 Deafness, X-linked Hereditary deafness is divided into syndromic forms (in which hearing loss is associated with a variety of other anomalies) and non-syndromic forms. Non-syndromic forms are responsible for 70% of the cases ... Nervous system disease
H01210 Hypomagnesemia Hypomagnesemia (HOMG) is defined as a serum magnesium level less than 1.8 mg/dl. Hypomagnesemia may result from inadequate magnesium intake, increased gastrointestinal or renal losses, or redistribution ... Inherited metabolic disorder
H01211 MECP2-related severe neonatal encephalopathy MECP2-related severe neonatal encephalopathy is a rare disorder of males characterized by a static encephalopathy, severe developmental delays, hypotonia, seizures, and respiratory abnormalities that often ... Congenital malformation
H01212 Familial encephalopathy with neuroserpin inclusion bodies Familial encephalopathy with neuroserpin inclusion bodies (FEBIB) is an autosomal dominant dementia that is characterized by the accumulation of mutant neuroserpin as periodic acid Schiff (PAS) positive ... Neurodegenerative disease
H01213 Gallbladder disease Gallbladder disease is one of the most common digestive disorders. Cholecystitis is an inflammation of the gallbladder wall, that is almost always associated with cholelithiasis, or gallstones. Cholecystitis ... Digestive system disease
H01214 Rh-null hemolytic anemia (RHN)
Rh-deficiency syndrome
Rh (Rhesus) null hemolytic anemia (RHN) is a rare autosomal recessive disorder characterized by an absence of Rh antigens and a varying degree of hemolytic anemia and spherostomatocytosis. The Rh protein ... Hematologic disease
H01215 Simpson-Golabi-Behmel syndrome Simpson Golabi Behmel syndrome (SGBS) is a complex congenital overgrowth syndrome with features that include macroglossia, macrosomia, and renal and skeletal abnormalities as well as an increased risk ... Congenital malformation
H01216 Left ventricular noncompaction Left ventricular noncompaction (LVNC) is a rare and potentially progressive cardiomyopathy, characterized by the presence of prominent trabeculations of the left ventricle, associated with progressive ... Cardiovascular disease
H01217 Primary localized cutaneous amyloidosis Primary localized cutaneous amyloidosis (PLCA) is a chronic itchy skin disorder associated with amyloid deposits in the superficial dermis. It is a purely cutaneous disease with no association with systemic ... Skin disease
H01218 P14 deficiency The deficiency of the late endosomal-lysosomal MEK binding partner 1 (MP1)-interacting protein (also known as p14 and MAPBPIP) causes a primary immunodeficiency syndrome comprising congenital neutropenia ... Primary immunodeficiency
H01219 Restrictive cardiomyopathy Restrictive cardiomyopathy (RCM) is an uncommon heart muscle disorder characterized by impaired ventricular filling and increased stiffness of the myocardium with diastolic dysfunction, resulting in atrial ... Cardiovascular disease
H01220 Congenital cataracts, facial dysmorphism, and neuropathy Congenital cataracts, facial dysmorphism, and neuropathy (CCFDN) syndrome is a rare autosomal recessive, complex developmental disorder exclusively manifested in the Roma population. CCFDN is a genetically ... Congenital malformation
H01221 Epithelial basement membrane corneal dystrophy
Cogan microcystic epithelial dystrophy
Map-dot-fingerprint dystrophy
Epithelial basement membrane corneal dystrophy (EBMD) is a common bilateral epithelial dystrophy. There is usually no hereditary pattern, but some cases presented with an autosomal dominant inheritance ... Nervous system disease
H01222 Cowden syndrome Cowden syndrome (CS) is an autosomal dominant inherited cancer syndrome characterized by benign and malignant breast, thyroid, and endometrial neoplasias in addition to cutaneous findings and macrocephaly ... Congenital malformation
H01223 Mental retardation-stereotypic movements-epilepsy and/or cerebral malformations
Chromosome 5q14.3 deletion syndrome
Mental retardation-stereotypic movements-epilepsy and/or cerebral malformations (MRSME) is a disorder characterized by severe mental retardation, absent speech, hypotonia, poor eye contact and stereotypic ... Chromosomal abnormality
H01224 Ketosis-prone diabetes mellitus Ketosis-prone diabetes (KPD) is a widespread, emerging, heterogeneous syndrome, mostly observed in subjects of west African origin (west Africans and African-Americans), characterized by patients who present ... Endocrine and metabolic disease
H01225 D-2-hydroxyglutaric aciduria D-2-hydroxyglutaric aciduria (D-2-HGA) is an autosomal recessive neurometabolic disorder. Clinical symptoms of D-2-HGA are developmental delay, epilepsy, hypotonia, and dysmorphic features. It has been ... Inherited metabolic disorder
H01226 Polysyndactyly
Preaxial polydactyly type IV
Polysyndactyly, also known as preaxial polydactyly type IV, is a rare autosomal dominant limb malformation, caused by mutations in the GLI3 gene. It is comprising duplicated halluces, with syndactyly of ... Congenital malformation
H01227 Inflammatory bowel disease (IBD) Inflammatory bowel disease (IBD) is a heterogeneous group of chronic disorders, which includes Crohn's disease and ulcerative colitis. Patients suffer chronically from abdominal pain, diarrhea, bleeding ... Immune system disease hsa05321 Inflammatory bowel disease
H01228 Insulin-resistant diabetes mellitus with acanthosis nigricans
Type A insulin resistance
Insulin-resistant diabetes mellitus with acanthosis nigricans (IRAN) is an unusual cause of diabetes that result from metabolic abnormalities associated with mutations of the insulin receptor (INSR) gene ... Metabolic disease; Endocrine disease
H01229 Inclusion body myopathy 3 Inclusion body myopathy 3 (IBM3) is an autosomal dominant myopathy associated with a heterozygous missense mutation in the myosin heavy chain (MyHC) IIa gene (MYH2), changing the highly conserved and negatively ... Nervous system disease; Musculoskeletal disease
H01230 Adult-onset autosomal dominant leukodystrophy Adult-onset autosomal dominant leukodystrophy (ADLD) is a slowly progressive neurological disorder characterized by symmetrical widespread myelin loss in the central nervous system. Although a phenotype ... Nervous system disease
H01231 Biotin-thiamine-responsive basal ganglia disease
Thiamine metabolism dysfunction syndrome 2
Biotin-thiamine-responsive basal ganglia disease (BTBGD) is a rare autosomal recessive disorder. The disease has its onset in childhood, and is characterized by episodic encephalopathy presenting as confusion ... Inherited metabolic disorder
H01232 Syndromic multisystem autoimmune disease Syndromic multisystem autoimmune disease is a group of hereditary disorders that have a number of clinical features in addition to autoimmune disease. For example, ADMFD is characterized by organomegaly ... Immune system disease
H01233 Urocanase deficiency
Urocanic aciduria
Urocanase deficiency is an autosomal recessive disorder of histidine metabolism caused by mutations in the UROC1 gene. It is characterized by mental retardation, urocanic aciduria, and a defective activity ... Inherited metabolic disorder
H01234 Trimethylaminuria
Fish-odor syndrome
Trimethylaminuria (TMAU), also known as fish odour syndrome, is a metabolic disorder. TMAU usually presents with a body odour resembling that of rotten or decaying fish, the result of excess excretion ... Inherited metabolic disorder
H01235 Bleeding disorder platelet-type Bleeding disorder platelet-type is a condition characterized by mild to moderate mucocutaneous bleeding. Patients are with platelet dysfunction but normal platelet number. It has been reported that these ... Hematologic disease
H01236 Familial articular chondrocalcinosis Chondrocalcinosis is a common cause of joint pain and arthritis caused by calcium deposition in articular cartilage and the presence of calcium hypophosphate crystals in synovial fluid, cartilage and periarticular ... Musculoskeletal disease
H01237 Sulfite oxidase deficiency Sulfite oxidase deficiency is a rare inherited disease with severe neurological symptoms such as intractable neonatal or infantile seizures, hypotonia or hypertonia, feeding difficulties, profound developmental ... Inherited metabolic disorder
H01238 Phelan-McDermid syndrome
Chromosome 22q13.3 deletion syndrome
Phelan-McDermid syndrome is a genetic disorder caused by a microdeletion on chromosome 22. It is characterized by severe expressive-language delay, mild mental retardation, hypotonia, joint laxity, dolichocephaly ... Chromosomal abnormality
H01239 Combined SAP deficiency
Prosaposin deficiency
Combined SAP deficiency is a fatal infantile lysosomal storage disorder with hepatosplenomegaly and severe neurological disease. It is caused by mutations in the PSAP gene leading to absence or non-functionality ... Inherited metabolic disorder, Lysosomal disease
H01240 Immune thrombocytopenia
Autoimmune thrombocytopenic purpura
Immune thrombocytopenic purpura (ITP) is an acquired autoimmune disorder, and the most common cause of isolated thrombocytopenia in children. Destruction of autoantibody-sensitized platelets by Fc[gamma]R-bearing ... Immune system disease
H01241 Congenital diaphragmatic hernia Congenital diaphragmatic hernia (CDH) is defined as a protrusion of abdominal viscera into the thorax through an abnormal opening or defect that is present at birth. CDH is often associated with potentially ... Congenital malformation
H01242 Saccharopinuria Saccharopinuria is a metabolic disorder caused by a defect in a bifunctional protein with lysine-ketoglutarate reductase (LKR) activity and saccharopine dehydrogenase (SDH) activity, aminoadipic semialdehyde ... Inherited metabolic disorder
H01243 Huntington disease-like syndrome Huntington disease (HD), which is caused by a triplet-repeat expansion in the IT15 gene (also known as huntingtin or HD), accounts for about 90% of cases of chorea of genetic etiology. In recent years ... Neurodegenerative disease
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