First Potential Gene Therapy for Tay-Sachs Disease Produces Positive Results at Three Month Mark

Tay-Sachs disease is a rare, genetic and sadly fatal condition. It's a neurodegenerative disorder caused by an impaired production of the β-Hexosaminidase A enzyme (HEXA). Symptoms of this condition include listlessness, diminishing…

Continue Reading First Potential Gene Therapy for Tay-Sachs Disease Produces Positive Results at Three Month Mark
Potential Gene Therapies for Tay-Sachs, Sandhoff, and GM1 Gangliosidosis Now Have Worldwide Licenses
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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…

Continue Reading Potential Gene Therapies for Tay-Sachs, Sandhoff, and GM1 Gangliosidosis Now Have Worldwide Licenses
New Expanded Access Trials For FENIB and LOTS Patients Set to Begin
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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…

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