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Published: 20 August 2026

An Important Milestone for the GM2 Community - Taken From NTSAD

An Important Milestone for the GM2 Community The U.S. Food and Drug Administration (FDA) has authorized a University of Massachusetts (UMass) Chan Medical School research team to proceed with a...

Published: 17 June 2026

About Nizubaglustat

Nizubaglustat About Nizubaglustat Nizubaglustat (AZ-3102): a new investigational drug for GM2 gangliosidosis and other rare diseases Short summary Nizubaglustat (AZ-3102) is an experimental drug...

Published: 17 June 2026

NAVIGATE: a phase 3 international clinical trial for patients with NPCs and GM1/GM2 gangliosidos

NAVIGATE: a phase 3 international clinical trial for patients with NPCs and GM1/GM2 gangliosidos Patients with Niemann-Pick disease type C (NPC) and GM1 or GM2 gangliosidos may be enrolled in the...

Published: 17 August 2026

We have established cooperation with a patient organization in Slovakia

We are pleased to announce that we have established cooperation with the Slovak patient organization Civic Association Tay-Sachs SK, which supports patients with Tay-Sachs disease and other forms of...

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