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Entry Name Description Category Pathway
H02448 Harel-Yoon syndrome Harel-Yoon syndrome (HAYOS) is a neurological syndrome characterized by global developmental delay, hypotonia, optic atrophy, axonal neuropathy, and hypertrophic cardiomyopathy. It has been reported that ... Nervous system disease
H02449 Autosomal dominant lamellar ichthyosis Autosomal dominant lamellar ichthyosis (ADLI) is a skin disorder caused by mutations in ASPRV1. Clinical and histologic features are similar to those of autosomal-recessive lamellar ichthyosis [DS:H00734] ... Congenital malformation
H02450 Horizontal gaze palsy with progressive scoliosis Horizontal gaze palsy with progressive scoliosis (HGPPS) is an autosomal recessive disorder characterized by congenital absence of horizontal conjugate eye movements with progressive scoliosis developing ... Nervous system disease
H02451 Congenital disorder of glycosylation with defective fucosylation Congenital disorder of glycosylation with defective fucosylation (CDGF) is a rare inherited metabolic disorder due to abnormalities in fucosylation, which is an enzymatic process of incorporating L-fucose ... Inherited metabolic disorder
H02452 Muscle hypertrophy Muscle hypertrophy (MSLHP) is defined as an increase in muscle mass, as a result of an increase in the size of pre-existing muscle fibers. A myostatin mutation in a child with muscle hypertrophy has been ... Musculoskeletal disease
H02453 Congenital heart defects and ectodermal dysplasia Congenital heart defects and ectodermal dysplasia (CHDED) is one of the syndromic congenital heart defects. Patients have extra-cardiac manifestations, including severe developmental delay, ectodermal ... Congenital malformation
H02454 Congenital heart defects, dysmorphic facial features, and intellectual developmental disorder Congenital heart defects, dysmorphic facial features, and intellectual developmental disorder (CHDFIDD) is one of the syndromic congenital heart defects. It has been reported that heterozygous mutations ... Congenital malformation
H02455 Congenital heart defects, hamartomas of tongue, and polysyndactyly
Ostravik-Lindemann-Solberg syndrome
Congenital heart defects, hamartomas of tongue, and polysyndactyly (CHDTHP), also known as Ostravik-Lindemann-Solberg syndrome, is a rare, autosomal recessive disorder characterized by coarctation of the ... Congenital malformation
H02456 Ectodermal dysplasia Ectodermal dysplasias (ECTD) are a heterogeneous group of disorders characterized by a deficiency of ectodermally derived tissues, including hair, skin, teeth, and sweat glands. These conditions feature ... Congenital malformation
H02457 Developmental delay, leukoencephalopathy, and neurologic decompensation Developmental delay, leukoencephalopathy, and neurologic decompensation is an autosomal dominant neurodevelopmental syndrome caused by missense mutations in EIF2AK1 and EIF2AK2. They encode members of ... Nervous system disease
H02458 Hydrocephalus due to congenital stenosis of aqueduct of Sylvius
X-linked hydrocephalus
Hydrocephalus due to congenital stenosis of aqueduct of Sylvius (HSAS) is a rare X-linked disease characterized by severe hydrocephaly. HSAS is the most common of the inherited form of hydrocephalus. It ... Congenital malformation
H02459 Syndromic neurodevelopmental disorder Syndromic neurodevelopmental disorder (NED) is a group of disorders that have a number of clinical features in addition to intellectual disability and developmental delay. Many genes that cause these syndromes ... Congenital malformation
H02460 Neurodevelopmental disorder with dysmorphic facies and skeletal anomalies Neurodevelopmental disorder (NED) with dysmorphic facies and skeletal anomalies is a group of syndromic neurodevelopmental disorders. Some of them have complications in addition to dysmorphic facies and ... Congenital malformation
H02461 Neurodevelopmental disorder with microcephaly Neurodevelopmental disorder (NED) with microcephaly is a group of syndromic neurodevelopmental disorders. Most of them have complications in addition to microcephaly. Several underlying genetic causes ... Congenital malformation
H02462 Spondyloepiphyseal dysplasia Spondyloepiphyseal dysplasia (SED) refers to a heterogeneous group of disorders with primary involvement of vertebrae and epiphyseal centers of long bones. Three major types of SED are recognized SED congenita ... Congenital malformation
H02463 Syndromic intellectual developmental disorder Syndromic intellectual developmental disorder (IDD) is a group of disorders characterized by intellectual disability presented together with additional features such as facial dysmorphisms or congenital ... Mental and behavioural disorder
H02464 Poretti-Boltshauser syndrome Poretti-Boltshauser syndrome (PTBHS) is an autosomal recessive disorder characterized by nonprogressive cerebellar ataxia, intellectual disability, and ocular apraxia with cerebellar cysts. It has been ... Congenital malformation
H02465 Weiss-Kruszka syndrome Weiss-Kruszka syndrome is an autosomal dominant disorder characterized by craniofacial anomalies, corpus callosum dysgenesis, ptosis, and developmental delay. It has been reported that haploinsufficiency ... Congenital malformation
H02466 Rajab interstitial lung disease with brain calcification Rajab interstitial lung disease with brain calcification (RILDBC) is severe growth restriction with combined brain, liver and lung involvement. Mutations in genes encoding phenylalanyl-tRNA synthetases ... Respiratory system disease
H02467 Neonatal inflammatory skin and bowel disease Neonatal inflammatory skin and bowel disease (NISBD) is a rare autosomal recessive disorder characterized by inflammatory features with neonatal onset, involving the skin, hair, and gut. A few gene mutations ... Immune system disease
H02468 Early childhood-onset progressive leukodystrophy Early childhood-onset progressive leukodystrophy (PLDECO) is a rare autosomal recessive disorder caused by deficiency of the alkaline ceramidase. Mutations in the ACER3 gene result in inactivation of ACER3 ... Inherited metabolic disorder
H02469 Cone-rod synaptic disorder Cone-rod synaptic disorder (CRSD) is an autosomal recessive disorder caused by mutations in CABP4. CRSD is characterized by congenital nystagmus, stable low vision, photophobia, and a normal fundus appearance ... Nervous system disease
H02470 Neurodevelopmental disorder with structural brain abnormalities Neurodevelopmental disorder (NED) with structural brain abnormalities is a group of syndromic neurodevelopmental disorders. Most of them have complications in addition to microcephaly. Several underlying ... Congenital malformation
H02471 Brain malformations with urinary tract defects Brain malformations with urinary tract defects (BRMUTD) is a novel human syndrome associated with 1p32-p31 deletion involving the NFIA gene. NFIA encodes a member of the NFI family of transcription factors Congenital malformation
H02472 Early-onset progressive encephalopathy Early-onset progressive encephalopathy with brain atrophy and spasticity (PEBAS) is caused by mutations in TRAPPC12. Changes in Golgi morphology, membrane trafficking dysfunction, and mitotic delay were ... Nervous system disease
H02473 Leukoencephalopathy, brain calcifications, and cysts
Labrune syndrome
Leukoencephalopathy, brain calcifications, and cysts (LCC) is a rare disorder that is radiologically characterized by edematous leukoencephalopathy, cerebral calcifications, and formation of parenchymal ... Nervous system disease
H02474 Blepharocheilodontic syndrome Blepharocheilodontic syndrome (BCDS) is a rare autosomal dominant disorder characterized by eyelid malformations, cleft lip/palate, and ectodermal dysplasia. It has been reported that BCDS is caused by ... Congenital malformation
H02475 Retinoschisis Retinoschisis (RS) is a retinal disorder with macular degeneration. In most cases, juvenile retinoschisis is transmitted as an X-linked recessive trait. The RS1 gene responsible for X-linked juvenile retinoschisis ... Congenital malformation
H02476 Childhood-onset neurodegeneration with brain atrophy Childhood-onset neurodegeneration with brain atrophy (CONDBA) is an autosomal dominant disorder characterized by developmental regression and neurodegeneration in childhood. It has been reported that mutations ... Nervous system disease
H02477 Cohen-Gibson syndrome Cohen-Gibson syndrome (COGIS) is an overgrowth syndrome caused by mutations in EED. Patients are with overgrowth, facial dysmorphism and intellectual disability. EED is a member of the polycomb repressive ... Congenital malformation
H02478 CATIFA syndrome CATIFA syndrome is a novel autosomal recessive syndrome characterized by cleft lip, cataract, tooth abnormality, intellectual disability, facial dysmorphism, and ADHD. It has been reported that mutations ... Congenital malformation
H02479 Nivelon-Nivelon-Mabille syndrome Nivelon-Nivelon-Mabille syndrome (NNMS), previously known as chondrodysplasia-pseudohermaphrodism syndrome, is a rare autosomal recessive syndrome characterized by microcephaly, early infantile onset seizures ... Congenital malformation
H02480 Fontaine progeroid syndrome Fontaine progeroid syndrome (FPS) is a rare genetic disorder characterized by early aging, bone dysplasia, characteristic face, and early demise. Mutations in SLC25A24, that encodes for calcium-binding ... Congenital malformation
H02481 Syndromic disorder with short stature Short stature is one of the major components of many dysmorphic syndromes. Growth failure may be due to a wide variety of mechanisms, either related to the growth hormone (GH) or to underlying unknown ... Congenital malformation
H02482 ROSAH syndrome ROSAH syndrome is an autosomal dominant systemic disorder characterized by retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis, and migraine headache. It has been reported that mutations in ... Congenital malformation
H02483 Basel-Vanagaite-Smirin-Yosef syndrome Basel-Vanagaite-Smirin-Yosef syndrome (BVSYS) is a syndrome characterized by eye, brain, cardiac and palatal abnormalities as well as growth retardation, microcephaly and severe intellectual disability ... Congenital malformation
H02484 X-linked reticulate pigmentary disorder with systemic manifestations X-linked reticulate pigmentary disorder with systemic manifestations (XLPDR), previously known as X-linked cutaneous amyloidosis, is a rare syndrome characterized by recurrent infections and sterile multiorgan ... Immune system disease
H02485 Extraoral halitosis due to MTO deficiency Extraoral halitosis due to MTO deficiency (EHMTO) is a novel inborn error of metabolism. Mutations in SELENBP1 in patients with cabbage-like breath odor have been identified. The malodor was attributable ... Inherited metabolic disorder
H02486 HELIX syndrome HELIX syndrome is characterized by hypohidrosis, electrolyte imbalance, lacrimal gland dysfunction, ichthyosis, and xerostomia. It has been reported that mutations in CLDN10 cause HELIX syndrome. Skin disease
H02487 Diencephalic-mesencephalic junction dysplasia syndrome Diencephalic-mesencephalic junction dysplasia syndrome (DMJDS) is a novel autosomal recessive brain malformation. DMJDS is associated with a characteristic butterfly-shaped contour of the midbrain on axial ... Congenital malformation
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