PARAMUS, NJ, Feb. 27, 2026 (GLOBE NEWSWIRE) -- Polaryx Therapeutics (Nasdaq: PLYX), a clinical-stage biotechnology company developing novel, disease-modifying therapies for rare, pediatric lysosomal ...

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.

Lysosomal Storage Disorders are genetic conditions caused by enzyme deficiencies that lead to harmful substance buildup in cells, resulting in progressive organ damage. Common disorders in India ...

UAB is participating in a Phase I/II trial of AMT 191, a one time gene therapy designed to enable patients with classic Fabry disease to produce their own missing enzyme, potentially reducing the need ...

Regulatory milestone supports advancement of the SOTERIA Phase 2 basket trial evaluating PLX‑200 across multiple lysosomal storage disorders PARAMUS, NJ, March 17, 2026 (GLOBE NEWSWIRE) -- Polaryx ...

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.

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 ...

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 ...

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 ...