Gene Therapy Appears to Stabilize Symptoms in Infant Tay-Sachs Disease Patient
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Gene Therapy Appears to Stabilize Symptoms in Infant Tay-Sachs Disease Patient

According to a story from WBUR, a preliminary evaluation of a young patients diagnosed with Tay-Sachs disease appears to show some encouraging signs. Two patients were treated with an experimental…

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In Honor of Tay-Sachs Disease Awareness Month Organizations are Teaming Together to Spread Awareness of Genetic Testing

Tay-Sachs Disease September is Tay-Sachs Disease Awareness Month. Voted on by the Senate unanimously in 2008, this month aims to spread awareness about this disease and the importance of carrier…

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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…

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$1.4 Million Donated to Help Bring a Gene Therapy For Tay-Sachs Disease to Clinical Trial
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$1.4 Million Donated to Help Bring a Gene Therapy For Tay-Sachs Disease to Clinical Trial

A $1.4 million dollar donation to the University of Massachusetts Medical School will help to fund research into a gene therapy for Tay-Sachs disease as it transitions from pre-clinical to…

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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…

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Photographer Captures Daily Life of Rare Families with Dignity and Grace
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Photographer Captures Daily Life of Rare Families with Dignity and Grace

Rare disease began shaping acclaimed photographer, Karen Haberberg's family years before she was born. Her parents had given birth to her older brother, who was diagnosed with Tay-Sachs. Tay-Sachs is a…

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