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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Patient association
Tay-Sachs SK
We are here for patients with Tay-Sachs disease and other forms of GM2 gangliosidosis, and for their families in Slovakia.
Why we exist
Our mission
We are a non-profit organisation founded so that patients with the rare Tay-Sachs disease and their families do not have to face their situation alone.
- We support patients with Tay-Sachs disease and other forms of GM2 gangliosidosis, and their families.
- We bring together patients, their loved ones and family members.
- We raise public awareness of this rare disease.
- We cooperate with doctors, researchers and organisations abroad.
- We help secure treatment, rehabilitation and social support.
- We protect the rights and interests of patients.
How you can support us
Any contribution helps, large or small — we use it directly to support patients and their families. If you would like to support us financially, we will be very grateful.
Account number (IBAN)
SK31 1100 0000 0029 4803 7720
All information about supporting us
News
All news
Latest news
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.
Read moreAbout 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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