Lysosomal Storage Diseases D016464

Description

Inborn errors of metabolism characterized by defects in specific lysosomal hydrolases and resulting in intracellular accumulation of unmetabolized substrates.   MeSH

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Subtype Terms (7)

Aspartylglucosaminuria
9 drugs (7 approved, 2 experimental)

Cholesterol Ester Storage Disease
1 approved drug

Cystinosis
7 drugs (4 approved, 3 experimental)

Lysosomal Storage Diseases, Nervous System
 

Mannosidase Deficiency Diseases
5 drugs (4 approved, 1 experimental)

Mucopolysaccharidoses
33 drugs (15 approved, 18 experimental)


Phase 3 Indicated Drugs (3)

Phase 2 Indicated Drugs (2)


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UMLS Data


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