familial mucolipidosis
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MONDO_0031422 |
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pseudoachondroplasia
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MONDO_0008322 |
[Pseudoachondroplasia is characterized by severe growth deficiency and deformations such as bow legs and hyperlordosis.] |
peripheral vascular disease
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MONDO_0005294 |
[Any disorder affecting blood flow through the veins or arteries outside of the heart.] |
GNPTG-mucolipidosis
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MONDO_0009652 |
[A very rare lysosomal disease, that has most often been observed in the Middle East, characterized by a progressive slowing of the growth rate in early childhood; stiffness and pain in shoulders, hips, and finger joints; a gradual, mild coarsening of facial features; and by a slower progression, milder clinical course and longer life expectancy than that seen in mucolipidosis II and mucolipidosis III alpha/beta. Cognitive function is normal or only slightly impaired and retinitis pigmentosa has been reported in a few patients. Many survive into early adulthood, but ultimately succumb to cardiorespiratory insufficiency.] |
Menkes disease
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MONDO_0010651 |
[A usually severe multisystemic disorder of copper metabolism, characterized by progressive neurodegeneration and marked connective tissue anomalies as well as typical sparse abnormal steely hair.] |
disorder of copper metabolism
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MONDO_0017762 |
[An inherited metabolic disease that is has its basis in the disruption of cellular copper ion homeostasis.] |
Renpenning syndrome
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MONDO_0010653 |
[An X-linked syndrome characterized by intellectual deficiency, microcephaly, leanness and mild short stature.] |
Crouzon syndrome-acanthosis nigricans syndrome
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MONDO_0012833 |
[Crouzon syndrome with acanthosis nigricans (CAN) is a very rare, clinically heterogeneous form of faciocraniostenosis with Crouzon-like features and premature synostosis of cranial sutures (Crouzon disease), associated with acanthosis nigricans (AN).] |
oculocutaneous albinism type 1
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MONDO_0018135 |
[Type 1 oculocutaneous albinism (OCA1) describes a group of tyrosine related OCAs that includes OCA1A, OCA1B, type 1 minimal pigment oculocutaneous albinism (OCA1-MP) and type 1 temperature sensitive oculocutaneous albinism (OCA1-TS).] |
antecubital pterygium syndrome
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MONDO_0008339 |
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dermis disorder
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MONDO_0021154 |
[A disease that involves the dermis.] |
oculocerebrorenal syndrome
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MONDO_0010645 |
[Oculocerebrorenal syndrome of Lowe (OCRL) is a multisystem disorder characterized by congenital cataracts, glaucoma, intellectual disabilities, postnatal growth retardation and renal tubular dysfunction with chronic renal failure.] |
holocarboxylase synthetase deficiency
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MONDO_0009666 |
[A life-threatening early-onset form of multiple carboxylase deficiency, an inborn error of biotin metabolism, that, if untreated, is characterized by vomiting, tachypnea, irritability, lethargy, exfoliative dermatitis, and seizures that can worsen to coma.] |
metabolic epilepsy
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MONDO_0100033 |
[Metabolic epilepsies are conceptualized as having a distinct metabolic abnormality that has been demonstrated to be associated with a substantially increased risk of developing epilepsy in appropriately designed studies. Metabolic disorders have genetic origin; however, the metabolic abnormalities are a separate disorder interposed between the genetic defect and the epilepsy.] |
biotinidase deficiency
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MONDO_0009665 |
[Biotinidase deficiency is a late-onset form of multiple carboxylase deficiency, an inborn error of biotin metabolism that, if untreated, is characterized by seizures, breathing difficulties, hypotonia, skin rash, alopecia, hearing loss and delayed development.] |
mulibrey nanism
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MONDO_0009664 |
[A prenatal onset growth disorder with multiorgan manifestations.] |
disorder of defective peroxisome oxidative status
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MONDO_0100306 |
[Any peroxisomal single enzyme/protein defect that has its basis in the disruption of peroxisome oxidation.] |
pseudo-von Willebrand disease
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MONDO_0008332 |
[A bleeding disorder characterized by mild to moderate mucocutaneous bleeding, which becomes more pronounced during pregnancy or following ingestion of drugs that have anti-platelet activity. PT-VWD is due to hyperresponsive platelets, resulting in thrombocytopenia.] |
hereditary von Willebrand disease
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MONDO_0019565 |
[Hereditary von Willebrand disease (VWD) is a hereditary bleeding disorder caused by a genetic anomaly leading to quantitative, structural or functional abnormalities of the Willebrand factor (von Willebrand factor; VWF). Two major groups of VWF deficiency have been defined: quantitative and partial (type 1) or total (type 3), and qualitative (type 2) with several subtypes (2A, 2B, 2M, 2N).] |
mucopolysaccharidosis type 7
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MONDO_0009662 |
[Mucopolysaccharidosis type VII (MPS VII) is a very rare lysosomal storage disease belonging to the group of mucopolysaccharidoses.] |