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Entry Name Description Category Pathway
H01284 Marinesco-Sjogren syndrome Marinesco-Sjogren syndrome is an autosomal recessive multisystem disorder which is characterized by cerebellar ataxia, progressive myopathy and cataracts. Mutations in the endoplasmic reticulum associated ... Nervous system disease
H01285 Methylcobalamin deficiency type G Methylcobalamin deficiency type G (cblG) is an autosomal recessive disease characterized by homocystinuria, hyperhomocysteinemia, and hypomethioninemia. Mutations in the MTR gene, which encodes methionine ... Inherited metabolic disorder
H01286 Microtia hearing impairment and cleft palate Microtia is a congenital anomaly of the ear characterized by a small abnormally shaped outer ear. It is often associated with hearing loss. Syndromic form of microtia occurs in conjunction with other abnormalities ... Congenital malformation
H01287 Congenital mirror movements Mirror movements (MRMV) are involuntary movements of one side of the body that mirror intentional movements on the opposite side. While mirror movements are occasionally found in young children, persistence ... Nervous system disease
H01288 Mosaic variegated aneuploidy syndrome Mosaic variegated aneuploidy syndrome (MVA) is a rare autosomal recessive disorder characterized by mosaic aneuploidies, diverse phenotypic abnormalities and predisposition to cancer. MVA is due to defective ... Chromosomal abnormality
H01289 Mulibrey nanism Mulibrey nanism is an autosomal recessive growth disorder characterized by prenatal-onset growth failure and heart disease involving constrictive pericarditis and restrictive cardiomyopathy. Mutations ... Congenital malformation
H01290 Acute recurrent myoglobinuria Acute recurrent myoglobinuria is caused by inborn errors of glycogenolysis, mitochondrial fatty acid beta-oxidation, and oxidative phosphorylation. It has been reported that mutations in LPIN1 cause recurrent ... Inherited metabolic disorder
H01291 Spheroid body myopathy
Myofibrillar myopathy 3
Spheroid body myopathy (SBM) is a rare autosomal dominant neuromuscular disorder characterized by slowly progressing proximal muscle weakness and dysarthric nasal speech. The unique feature noted pathologically ... Nervous system disease; Musculoskeletal disease
H01292 Nance-Horan syndrome Nance-Horan syndrome (NHS) is an X-linked developmental disorder characterized by congenital cataract, dental anomalies, facial dysmorphism and, in some cases, mental retardation. Mutations in NHS gene ... Congenital malformation
H01293 Narcolepsy Narcolepsy is a disabling sleep disorder characterized by irresistible excessive daytime sleepiness and cataplexy, a condition triggered by strong emotions leading to a sudden loss of muscle tone. Narcolepsy ... Nervous system disease
H01294 Nephrogenic syndrome of inappropriate antidiuresis Nephrogenic syndrome of inappropriate antidiuresis (NSIAD) is a disorder of water balance caused by gain of function mutation of arginine vasopressin receptor type 2 (AVPR2) resulting in free water reabsorption ... Endocrine and metabolic disease
H01295 Neurodegeneration due to cerebral folate transport deficiency Neurodegeneration due to cerebral folate transport deficiency is an autosomal recessive disorder of brain specific folate deficiency, characterized by severe developmental regression, movement disturbances ... Neurodegenerative disease
H01296 Hereditary neuropathy with liability to pressure palsies Hereditary neuropathy with liability to pressure palsy (HNPP) is a rare autosomal dominant peripheral neuropathy characterized by compressive focal neuropathies and an underlying sensorimotor demyelinative ... Nervous system disease
H01297 Neutral lipid storage disease with myopathy Neutral lipid storage disease with myopathy (NLSDM) comprises a heterogeneous group of autosomal recessive disorders characterized by systemic accumulation of triglycerides in cytoplasmic droplets. Neutral ... Inherited metabolic disorder
H01298 Pulmonary alveolar microlithiasis Pulmonary alveolar microlithiasis (PALM) is a rare disease characterized by the deposition of calcium phosphate microliths throughout the lungs. Most patients are asymptomatic for several years, and cases ... Respiratory system disease
H01299 Idiopathic pulmonary fibrosis Idiopathic pulmonary fibrosis is a scarring lung disease that presents in older adults with shortness of breath and cough. Mutations in surfactant protein C (SFTPC), surfactant protein A (SFTPA), telomerase ... Respiratory system disease
H01301 Hemorrhagic destruction of the brain, subependymal calcification, and cataracts Hemorrhagic destruction of the brain, subependymal calcification, and cataracts (HDBSCC) is an autosomal-recessive syndrome with severe hemorrhagic destruction of the brain as a cardinal feature. A mutation ... Nervous system disease
H01302 Hyperchlorhidrosis isolated (HCHLH) Hyperchlorhidrosis is excessive chloride secretion in sweat. Abnormal sweat chloride levels is found also in conjunction with various metabolic, endocrine, and dermatological disorders. Hyperchlorhidrosis ... Inherited metabolic disorder
H01303 Hypercatabolic hypoproteinemia
Immunodeficiency 43
Hypercatabolic hypoproteinemia is a disorder of endogenous catabolism of albumin and immunoglobulin. FcRn is a heterodimeric receptor composed of a nonclassical MHC class I alpha-chain and beta-2-microglobulin ... Hematologic disease
H01304 Hyperglycinuria Hyperglycinuria (HG) is an autosomal recessive abnormality of renal transport of glycine, resulting from mutations in genes encoding proline and glycine transporters. Urinary system disease
H01305 Global cerebral hypomyelination
Early infantile epileptic encephalopathy 39
Global cerebral hypomyelination is a syndrome characterized by arrested psychomotor development, hypotonia, and seizures. A missense mutation in the SLC25A12, which encodes the AGC1 protein, causes this ... Inherited metabolic disorder, Mitochondrial disease
H01306 FRA12A mental retardation FRA12A mental retardation is a rare form of mental retardation caused by expansion of CGG repeat. This repeat is in the 5' untranslated region of the gene DIP2B. It has been suggested that deficiency of ... Mental and behavioural disorder
H01307 Nonsyndromic congenital nail disorder Nonsyndromic congenital nail disorder (NDNC) is rare and has been reported in only a small number of families. There is a variable expression of nail phenotypes among individuals. It has been identified ... Skin disease
H01308 Macrocephaly macrosomia facial dysmorphism syndrome Macrocephaly macrosomia facial dysmorphism syndrome is characterized by overgrowth, learning disability, and dysmorphic features. Mutations in RNF135 cause this syndrome. Congenital malformation
H01309 Sarcoidosis Sarcoidosis is a multisystem disease characterized by granulomatous inflammation throughout the body afflicting different organ systems. Pulmonary complaints are most common, however, cardiac, optic, and ... Immune system disease
H01310 Multi-minicore disease
Multicore myopathy with external ophthalmoplegia
Rigid spine syndrome (RSS)
Multi-minicore Disease (MmD) is a recessively inherited neuromuscular disorder characterized by multiple cores on muscle biopsy and clinical features of a congenital myopathy. It is morphologically defined ... Nervous system disease; Musculoskeletal disease
H01311 Enteroinvasive Escherichia coli (EIEC) infection Enteroinvasive Escherichia coli (EIEC) infection is common in developing countries and comparatively rare in developed countries. EIEC invades intestinal epithelial cells, causing necrosis, ulceration ... Bacterial infectious disease
H01312 Enteroaggregative Escherichia coli (EAEC) infection Enteroaggregative Escherichia coli (EAEC or EAggEC) infection is a cause of traveler's diarrhea and persistent watery diarrhea in young children and patients infected with HIV. EAEC adheres to the small ... Bacterial infectious disease
H01313 Escherichia coli meningitis
Neonatal meningitis-associated Escherichia coli (NMEC) infection
Escherichia coli meningitis is a bacterial meningitis caused by Escherichia coli, and is common in the newborn within the first month of life (neonatal meningitis). Neonatal meningitis-associated Escherichia ... Bacterial infectious disease
H01314 Rat-bite fever
Haverhill fever
Rat-bite fever is a systemic febrile zoonotic illness caused by either Streptobacillus moniliformis, common in Western countries, or Spirillum minus, which is the most prevalent pathogen in Asia. It is ... Bacterial infectious disease
H01315 Erysipeloid
Swine erysipelas
Erysipelothrix rhusiopathiae is a non-sporulating, gram-positive, rod-shaped bacterium which was identified as the etiologic agent of swine erysipelas. Infection in man is occupationally related, occurring ... Bacterial infectious disease
H01316 Dermatophytosis
Ringworm
Dermatophytosis (tinea, ringworm) is an infection of keratinized structures, such as the hair, skin or nails caused by dermatophyte fungi of the genera Trichophyton, Epidermophyton or Microsporum. Tinea ... Fungal infectious disease
H01317 Aggressive periodontitis Aggressive periodontitis is a disease of the periodontium occurring in an otherwise healthy adolescent, which is characterized by a rapid loss of alveolar bone around more than one tooth of the permanent ... Bacterial infectious disease
H01318 Yaws Treponemal infections occurring in humans comprise venereal syphilis and the non-venereal endemic treponematoses. The non-venereal endemic treponematoses are yaws, pinta, and endemic syphilis. Yaws is ... Bacterial infectious disease
H01319 Coccidioidomycosis
Valley fever
Coccidioides immitis and C. posadasii are two endemic dimorphic fungal pathogens that cause coccidioidomycosis, also called valley fever, and are found in the southwestern United States and northern Mexico ... Fungal infectious disease
H01320 Epidemic keratoconjunctivitis Epidemic keratoconjunctivitis (EKC) is a highly contagious and severe form of eye disease caused by human adenoviruses (HAdVs). There are two well-defined adenoviral keratoconjunctivitis clinical syndromes: ... Viral infectious disease
H01321 Pneumococcal disease Pneumococcal disease (PD) is infectious disease caused by Streptococcus pneumoniae (pneumococcus). PD is particularly common in younger children and in older adults and is roughly divided into invasive ... Bacterial infectious disease
H01322 Kyasanur Forest disease Kyasanur Forest disease is a viral hemorrhagic fever caused by Kyasanur Forest disease virus (KFDV), a flavivirus in the Flaviviridae family of +ssRNA viruses, and transmitted by Ixodoidea ticks. KFDV ... Viral infectious disease
H01323 Enteritis due to Norovirus Enteritis due to Norovirus is a major nonbacterial gastroenteritis worldwide. Norovirus is one of five genera of the family Caliciviridae of +ssRNA viruses. Human norovirus, previously known as Norwalk ... Viral infectious disease
H01324 Lymphocytic choriomeningitis Lymphocytic choriomeningitis (LCM) is an infectious disease caused by Lymphocytic choriomeningitis virus (LCMV), an arenavirus in the Arenaviridae family of -ssRNA viruses, and transmitted by rodents. ... Viral infectious disease
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