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.

Please provide your email address to receive an email when new articles are posted on . An Italian pharmaceutical company and a Boston-area biotechnology research firm announced a partnership to ...

This AJMC ® Profiles in Care focuses on payer challenges and strategies involved when managing treatments for lysosomal storage diseases and other rare diseases. Stay ahead of policy, cost, and ...

LONDON--(BUSINESS WIRE)--Technavio’s latest market research report on the global lysosomal storage diseases (LSDs) market provides an analysis of the most important trends expected to impact the ...

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

In the past week, two announcements from the drug discovery world have had Insilico Medicine’s generative artificial intelligence (AI) target discovery engine, PandaOmics, front and center. The first ...

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 ...

Polaryx Therapeutics, Inc. (Nasdaq: PLYX), a clinical-stage biotechnology company developing novel, disease-modifying therapies for rare, pediatric lysosomal storage disorders (“LSDs”), announces that ...

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 ...

A Denali treatment for a rare lysosomal storage disorder, Hunter syndrome, has posted encouraging biomarker results in a small, open-label Phase 1/2 trial. The genetic disorder is caused by mutations ...

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 ...