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Lysosomal Storage Diseases
Inborn errors of metabolism characterized by defects in specific lysosomal hydrolases and resulting in intracellular accumulation of unmetabolized substrates.
Subtype terms (7)
More specific conditions that are subtypes of Lysosomal Storage Diseases in the MeSH hierarchy.
- Aspartylglucosaminuria
9 drugs (7 approved, 2 experimental)
- Cholesterol Ester Storage Disease
1 drug approved
- Cystinosis
13 drugs (9 approved, 4 experimental)
- Lysosomal Storage Diseases, Nervous System
- Mannosidase Deficiency Diseases
5 drugs (4 approved, 1 experimental)
- Mucopolysaccharidoses
39 drugs (19 approved, 20 experimental)
- Pycnodysostosis
Drugs by status
Phase 3 trials (3)
Phase 2 trials (5)
Phase 1 trials (3)
Organizations
Organization Involved with Phase 3 Indications (5)
Organization Involved with Phase 2 Indications (7)
Organization Involved with Phase 1 Indications (4)
Organization Involved with Other Experimental Indications (7)