Lysosomal Storage Disorder Therapeutics – Analysis, Clinical Trials and Developments

Press Release

Lysosomal storage disorder therapeutics currently exhibits a proliferating pipeline with 75+ therapeutic candidates.

Lysosomal storage disorder refers to a group of genetically inherited disorders, which are characterized by enzyme deficiencies. It results in accumulation of toxic chemical substances in the body’s cells. This group of disorders consists of approximately 50 diseases, including Metachromatic leukodystrophy; Mucolipidosis types I, II/III, and IV; Mucopolysaccharide storage disease; Gaucher disease; Niemann-Pick disease type C (NPC); Schindler disease types I and II; Tay Sachs disease; Pompe disease; and Sandhoff disease. These diseases affect different body parts, including heart, bones, brain, and central nervous system (CNS). Mental retardation, hepatomegaly, absurd growth of facial bones, and optic atrophy are some of the most common symptoms reported in the disease.

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Currently, Ceredase, Aldurazyme, and Fabrazyme by Sanofi-aventis Groupe, and Zavesca by Actelion Pharmaceuticals Ltd. are some of the U.S. Food and Drug Administration (USFDA) approved drugs for the treatment of LSD. Moreover, due to emergence of late- and mid-stage pipeline products in the market, the overall LSD therapeutics market is expected to grow significantly in the upcoming years.

Orchard Therapeutics Limited, Sanofi-aventis Groupe, Actelion Pharmaceuticals Ltd., Amicus Therapeutics Inc., Takeda Pharmaceutical Company Limited, Ultragenyx Pharmaceutical Inc., Valerion Therapeutics LLC, Actus Therapeutics Inc., Chiesi Farmaceutici S.p.A., Resverlogix Corp., AVROBIO Inc., Mallinckrodt LLC, CTD Holdings Inc., Orphazyme A/S, Idorsia Pharmaceuticals Ltd., and Protalix BioTherapeutics Inc. are some of the key players involved in the development of therapeutic drugs indicated for the treatment of LSD.

LSD Therapeutics Pipeline Analysis

  • By Phase
  • By Molecule Type
  • By Route of Administration
  • By Company

This post was originally published on Financial Sector

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