Tay-Sachs SK

News

News from Tay-Sachs research, about treatment, and from the life of our association.

An important milestone for the GM2 community — via NTSAD

The US FDA has cleared the UMass Chan Medical School research team to proceed with a phase 1/2 clinical trial of a second-generation AAV gene therapy for GM2.

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We have established a partnership with the Czech patient organisation Taysachs

We are glad to announce that we have established a partnership with the Czech patient organisation Taysachs, which supports patients with Tay-Sachs disease and their families in the Czech Republic.

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About the drug nizubaglustat

Nizubaglustat (AZ-3102) is an experimental oral drug currently being studied as a possible treatment for certain rare inherited diseases affecting the nervous system.

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NAVIGATE: an international phase 3 clinical trial for patients with NPC and GM1/GM2 gangliosidosis

Patients with Niemann-Pick disease type C and GM1 or GM2 gangliosidosis can join the international phase 3 clinical trial NAVIGATE, sponsored by Azafaros.

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