Potential Gene Therapies for Tay-Sachs, Sandhoff, and GM1 Gangliosidosis Now Have Worldwide Licenses
Source: Pixabay.com

Potential Gene Therapies for Tay-Sachs, Sandhoff, and GM1 Gangliosidosis Now Have Worldwide Licenses

Tay-Sachs disease and Sandhoff disease (also known as GM2 Gangliosidosis), and GM1 Gangliosidosis are neurodegenerative conditions. They are a result of a defective enzyme. This enzyme causes toxic gangliosides to accumulate within the…

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New Expanded Access Trials For FENIB and LOTS Patients Set to Begin
<a href="https://pixabay.com/users/DarkoStojanovic/">DarkoStojanovic</a> / Pixabay

New Expanded Access Trials For FENIB and LOTS Patients Set to Begin

According to a story from globenewswire.com, the corporation Retrotope recently announced the initiation of single-patient, expanded access trials of its experimental product RT001. The patients involved have familial encephalopathy with neuroserpin…

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