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Tay-Sachs, Canavan, Sandhoff, GM1 and related diseases

FDA Approves Investigational Drug for Late Onset GM2

BREAKING NEWS: Clinical Trial Announced

After several years of collaboration and partnership at our annual family conferences - and many phone and in-person meetings with Sanofi Genzyme - we are very pleased to announce that the FDA approved the Investigational New Drug (IND) application submitted by Sanofi Genzyme for Venglustat.

Venglustat is a small molecule therapy for the Late Onset GM2 Gangliosidosis population.

In addition to the Late Onset GM2 study, there will also be a secondary arm of the study for Juvenile Onset GM1 and GM2 gangliosidosis and other rare disorders including saposin C deficiency, sialidosis type 1 and juvenile/adult galactosidosis.

The study is not yet recruiting as they are waiting for IRB approval at the clinical trial sites. Those will be announced as soon as we learn that they are officially open for recruitment.

For More Information

A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2 (AMETHIST) - Click here to read the full posting on clinicaltrials.gov.

Please contact This email address is being protected from spambots. You need JavaScript enabled to view it. at NTSAD with your questions.

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