The month of May is Huntington's Disease Awareness Month, in which a special effort is made to improve awareness about this devastating rare disease. This year, the Huntington's Disease Society…
Ionis Pharmaceuticals, in a partnership with Roche, has announced that the enrollment has been completed for the third phase of GENERATION HD1, a trial intended to study the safety and…
A two-part series of “The Gene”, a PBS documentary, will air on April 14, 2020. The film, by Ken Burns, is based on a book with the same title written…
A news release from Benzinga shares that biotechnology company Emerald Health Pharmaceuticals is beginning a Phase 2 clinical study on EHP-101 as a treatment for patients with systemic sclerosis.…
In a Special Issue of the journal Disease Models and Mechanisms Highlights, James J. Dowling et al describes pediatric neuromuscular diseases as mostly genetic and affecting areas of the peripheral…
As previously published in Scientific American, slightly after her first birthday, Emma Larson lost the use of her legs and started experiencing difficulty crawling. She was diagnosed with spinal muscular…
According to a recent article published in Pharmaceutical-Technology, uniQure’s gene therapy AMT-130 may lower levels of the huntingtin protein that causes the disorder. An abnormal gene is responsible for damage…
Current treatment for Huntington's disease only relieves symptoms, allowing for the progression of the disease to continue. UniQure, a company that works with gene therapies, aims to change this. They…
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