lysosomal storage disease
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MONDO_0002561 |
[A metabolic disorder caused by mutations in proteins critical for lysosomal function, including lysosomal enzymes, lysosomal integral membrane proteins, and proteins involved in the post-translational modification and trafficking of lysosomal proteins.] |
focal segmental glomerulosclerosis 9
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MONDO_0014539 |
[Any focal segmental glomerulosclerosis in which the cause of the disease is a mutation in the CRB2 gene.] |
inherited focal segmental glomerulosclerosis
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MONDO_0005363 |
[An instance of focal segmental glomerulosclerosis that is caused by an inherited genomic modification in an individual.] |
PIEZO1-related generalized lymphatic dysplasia with non-immune hydrops fetalis
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MONDO_0035474 |
[A rare genetic primary lymphedema characterized by uniform, widespread lymphedema, often with systemic involvement such as intestinal and pulmonary lymphangiectasia, pleural and pericardial effusions, and chylothorax. There is a high incidence of non-immune hydrops fetalis, which may result in fetal demise or fully resolve after birth. Severe, recurrent facial cellulitis is observed in some patients. Presence of epicanthic folds or micrognathia has occasionally been reported, while intelligence is normal, and seizures are absent.] |
primary lymphedema
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MONDO_0019175 |
[A congenital condition that results in swelling in the arms or legs, and can occur during adolescence or adulthood. Loss of motion and pain may also accompany the swelling. Protein-rich lymphatic fluid accumulates in tissues, engorging and enlarging vessels and often causing visible swelling, tenderness, and pain. Left untreated, the affected tissues may continue to swell, and can become hardened or fibrotic and susceptible to infection.] |
rhabdoid tumor predisposition syndrome 2
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MONDO_0013224 |
[Any familial rhabdoid tumor in which the cause of the disease is a mutation in the SMARCA4 gene.] |
atypical teratoid rhabdoid tumor
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MONDO_0020560 |
[Atypical teratoid rhabdoid tumor (ATRT) is a highly malignant central nervous system (CNS) rhabdoid tumor (RT) found almost exclusively in children.] |
familial rhabdoid tumor
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MONDO_0016473 |
[A neoplastic syndrome most often caused by mutations in the hSNF5/INI1 tumor suppressor gene. It is characterized by the development of an atypical teratoid/rhabdoid tumor in infancy and early childhood. This highly aggressive tumor develops in the central nervous system as an isolated lesion or in combination with extrarenal or renal rhabdoid tumor. Patients may also develop other central nervous system malignancies including medulloblastoma, supratentorial primitive neuroectodermal tumor, and choroid plexus carcinoma.] |
oculocutaneous albinism
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MONDO_0018910 |
[Oculocutaneous albinism (OCA) describes a group of inherited disorders of melanin biosynthesis characterized by a generalized reduction in pigmentation of hair, skin and eyes and variable ocular findings including nystagmus, reduced visual acuity and photophobia. Variants include OCA1A (the most severe form), OCA1B, OCA1-minimal pigment (OCA1-MP), OCA1-temperature sensitive (OCA1-TS), OCA2, OCA3, OCA4, OCA5, OCA6 and OCA7.] |
hypopigmentation of the skin
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MONDO_0019290 |
[A condition caused by a deficiency or a loss of melanin pigmentation in the epidermis, also known as hypomelanosis. Hypopigmentation can be localized or generalized, and may result from genetic defects, trauma, inflammation, or infections.] |
disorder of melanin metabolism
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MONDO_0018134 |
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congenital plasminogen activator inhibitor type 1 deficiency
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MONDO_0013227 |
[Congenital plasminogen activator inhibitor type 1 (PAI-1) deficiency is a rare genetic bleeding disorder characterized by premature lysis of hemostatic clots and a moderate bleeding tendency.] |
disorder of glycolysis
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MONDO_0017688 |
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monogenic diabetes
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MONDO_0015967 |
[Diabetes mellitus that is caused by mutations in a single gene.] |
Waardenburg syndrome type 4A
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MONDO_0010192 |
[A subtype of Waardenburg syndrome type 4 (Waardenburg-Shah syndrome) caused by mutations in EDNRB.] |
Weaver syndrome
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MONDO_0010193 |
[Weaver syndrome (WVS) is a rare, multisystem disorder characterized by tall stature, a typical facial appearance (hypertelorism, retrognathia) and variable intellectual disability. Additional features may include camptodactyly, soft doughy skin, umbilical hernia, and a low hoarse cry.] |
overgrowth syndrome
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MONDO_0019716 |
[A group of syndromes caused by genetic birth defects that may lead to the development of malignancies. It is characterized by a large body size or large body parts at birth, or excessive body growth early in childhood. Representative examples include neurofibromatosis, Beckwith-Wiedemann syndrome, and Sturge-Weber syndrome.] |
skeletal dysplasia
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MONDO_0018230 |
[Any Mendelian diseases that affects growth and development of the skeleton.] |
Werner syndrome
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MONDO_0010196 |
[A rare inherited syndrome characterized by premature aging with onset in the third decade of life and with cardinal clinical features including bilateral cataracts, short stature, graying and thinning of scalp hair, characteristic skin disorders and premature onset of additional age-related disorders.] |
progeroid syndrome
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MONDO_0015333 |
[A group of rare genetic disorders which mimic physiological aging, making affected individuals appear to be older than they are.] |