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 ...

Lysosomal storage diseases comprise a diverse group of inherited disorders in which deficient lysosomal hydrolases lead to the accumulation of undegraded substrates and subsequent cellular dysfunction ...

California this weekend will once again recognize a rare and deadly genetic disease that kills most of those affected between the ages of 2 and 4 — in large part because of a local couple who ...

Enzyme replacement therapy (ERT) has transformed the management of several lysosomal storage disorders by supplying patients with functional versions of the deficient hydrolases that accumulate ...

S03, a cell-based gene therapy designed to replace 130 lifetime Fabry infusions with a single procedure. FDA’s acceptance of INTERACT request validates the clinical and manufacturing case built on ...

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 ...

Company selected as a Diamond honoree for Excellence in Pediatric CarePARAMUS, NJ, April 15, 2026 (GLOBE NEWSWIRE) -- Polaryx Therapeutics (Nasdaq: PLYX), a clinical-stage biotechnology company ...

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 ...

Azafaros, a private company building a portfolio to become a leader in Lysosomal Storage Disorders and focused on addressing neurological symptoms, today announced that Amy Sullivan has joined the ...

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 ...