December 2, 2011 — Lysosomal storage disorders such as Fabry's disease and Pompe's disease are much more common than previously thought, particularly atypical later-onset forms, a new study suggests.
Glycogen storage diseases (GSDs) comprise a heterogeneous group of inherited metabolic disorders that impede the normal synthesis or degradation of glycogen, resulting in diverse clinical phenotypes.
Regulatory milestone supports advancement of the SOTERIA Phase 2 basket trial evaluating PLX‑200 across multiple lysosomal storage ...
The early funding will help transition its research into scalable therapies targeting lysosomal storage disorders.
McArdle disease, also known as glycogen storage disease type V, is an inherited metabolic disorder marked by a deficiency of myophosphorylase, the enzyme essential for the breakdown of glycogen in ...
Functioning brain cells need a functioning system for picking up the trash and sorting the recycling. But when lysosomes, the cellular sanitation machines responsible for those tasks, break down or ...
In a major breakthrough in the fight against rare diseases, a Gujarat-based institute has established India’s first national ...
HYOGO, Japan--(BUSINESS WIRE)--JCR Pharmaceuticals Co., Ltd. (TSE: 4552) made significant contributions at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium 2024, held ...
For Zevra Therapeutics and its rare neurodegenerative disease med arimoclomol, the second time’s the charm. Friday, the FDA approved Zevra’s arimoclomol capsules to treat the rare lysosomal storage ...
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India's first biobank for rare genetic disorders opens in Gujarat
India's first national biobank for rare Lysosomal Storage Disorders has been established in Ahmedabad, Gujarat. The ...
The RTI data highlights systemic shortcomings in how financial support is being delivered.
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