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.

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

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 ...

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 ...

HYOGO, Japan--(BUSINESS WIRE)--JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”), a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, today ...

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 ...

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 ...

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 ...

Funding supports IND-enabling activities for Casma’s CSM-101, a first-in-class TRPML1 agonist targeting lysosomal dysfunction in genetically driven neurodegenerative diseasesCAMBRIDGE, ...

Ahmedabad: The city recently got the national biobank for lysosomal storage disorders at the FRIGE Institute of Human Genetics. Lysosomal storage disorders (LSDs) are a diverse group of over 70 rare, ...

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 ...