Diseases [C] » Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] » Genetic Diseases, Inborn » Metabolism, Inborn Errors » Lysosomal Storage Diseases
Diseases [C] » Nutritional and Metabolic Diseases [C18] » Metabolic Diseases » Metabolism, Inborn Errors » Lysosomal Storage Diseases
Description
Inborn errors of metabolism characterized by defects in specific lysosomal hydrolases and resulting in intracellular accumulation of unmetabolized substrates. MeSH
Hierarchy View
Subtype Terms (7)
Aspartylglucosaminuria
9 drugs (7 approved, 2 experimental)
Cholesterol Ester Storage Disease
1 approved drug
Cystinosis
12 drugs (9 approved, 3 experimental)
Lysosomal Storage Diseases, Nervous System
Mannosidase Deficiency Diseases
5 drugs (4 approved, 1 experimental)
Mucopolysaccharidoses
39 drugs (19 approved, 20 experimental)
Phase 3 Indicated Drugs (3)
Phase 2 Indicated Drugs (1)
Phase 1 Indicated Drugs (3)
Other Experimental Indicated Drugs (8)
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)
Hierarchy Tree View
UMLS Data
YOU AGREE THAT THE INFORMATION PROVIDED ON THIS WEBSITE IS PROVIDED “AS IS”, WITHOUT ANY WARRANTY OF ANY KIND, EXPRESSED OR IMPLIED, INCLUDING WITHOUT LIMITATION WARRANTIES OF MERCHANTABILITY OR FITNESS FOR ANY PARTICULAR PURPOSE, OR NON-INFRINGEMENT OF ANY THIRD-PARTY PATENT, COPYRIGHT, OR ANY OTHER THIRD-PARTY RIGHT. IN NO EVENT SHALL THE CREATORS OF THE WEBSITE OR WASHINGTON UNIVERSITY BE LIABLE FOR ANY DIRECT, INDIRECT, SPECIAL, OR CONSEQUENTIAL DAMAGES ARISING OUT OF OR IN ANY WAY CONNECTED WITH THE WEBSITE, THE USE OF THE WEBSITE, OR THIS AGREEMENT, WHETHER IN BREACH OF CONTRACT, TORT OR OTHERWISE, EVEN IF SUCH PARTY IS ADVISED OF THE POSSIBILITY OF SUCH DAMAGES.
