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

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 ...

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 ...

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 ...

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 ...

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 ...

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 ...

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 ...

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 ...

Cholesteryl Ester Storage Disease (CESD) is a rare autosomal recessive multisystem condition that belongs to the lysosomal storage disorders family. The liver is one of the most common sites of ...

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.