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.

This new analysis shows early diagnosis of some lysosomal storage disorders can help avoid irreversible damage across the board. Lysosomal storage disorders (LSDs), including Gaucher disease (GD), ...

Main takeaways: Scientists at the Icahn School of Medicine at Mount Sinai in New York, the National Center for Advancing Translational Sciences (NCATS), and elsewhere have reversed the effects of ...

Gain Therapeutics, a preclinical biotech developing small molecule therapies for lysosomal storage disorders, announced terms for its IPO on Wednesday. The Bethesda, MD-based company plans to raise ...

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 ...

ST. LOUIS--(BUSINESS WIRE)--M6P Therapeutics (“M6PT” or “the Company”), a privately held life sciences company developing next-generation enzyme replacement and gene therapies for lysosomal storage ...

Lysosomal dysfunction is common in neurodegenerative disease. Childhood lysosomal storage disorders are poised for gene therapy trials. Can scientists learn from them to develop Alzheimer’s therapies?

Glycogen storage diseases (GSDs) constitute a group of inherited metabolic disorders that impair normal glycogen synthesis or degradation, resulting in the accumulation of structurally abnormal ...

One piece of legislation that could be on the state Legislature’s agenda next year is one that would require health insurers to provide coverage for continuous blood glucose monitors for children.

Polaryx is registering for sale approximately 179.8 million shares of its common stock, with the most recent private sale price being $0.64 per share since September 2025. No new institutional ...

ST. LOUIS--(BUSINESS WIRE)--M6P Therapeutics, a privately held life sciences company developing next-generation recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs), announced ...