Functioning brain cells need a functioning system for picking up the trash and sorting the recycling. But when the cellular ...

Together with colleagues from Stanford University, USA, researchers at the Leibniz Institute on Aging - Fritz Lipmann Institute (FLI) have, for the first time, created a comprehensive cell ...

Azafaros, a company building a portfolio to become a leader in lysosomal storage disorders with the goal of addressing neurological symptoms, today announced that its lead asset, nizubaglustat, will ...

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 ...

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 ...

Dr Sudheendra Rao N R from the Organization for Rare Diseases India reveals that approximately 70 million are affected by ...

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 ...

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 ...

Five presentations will showcase nizubaglustat's therapeutic potential in Lysosomal Storage Disorders. Nizubaglustat is currently being tested in two global Phase 3 studies for Niemann-Pick ...