Summary
- AMT-130 highlighted in Drug Discovery News
- Research Webinar Today: Hereditary Disease Foundation
- This week in HD History: Mutant Huntingtin Aggregates
AMT-130 highlighted in Drug Discovery News
HD research was put into the spotlight with a feature on uniQure’s experimental gene therapy for the treatment of Huntington’s disease, AMT-130, published last week in Drug Discovery News. The article covers the basic biology of the drug’s approach to treating HD, what we know about the safety and efficacy of uniQure’s approach so far, as well as perspectives from HD clinician, Dr. Ed Wild, and HDSA’s Assistant Director of Research & Patient Engagement, Dr. Leora Fox. To read the full article, click here.
Research Webinar Today: Hereditary Disease Foundation
Today, September 28th, at 12PM ET, the Hereditary Disease Foundation will host Drs. Judith Frydman and Natalia Barbosa from Stanford University for a Researcher Spotlight Webinar, “How Expanding Our Knowledge of the Huntington’s Protein Can Unlock New Therapies.” To register, click here.
This week in HD History: Mutant Huntingtin Aggregates
Huntington’s disease is caused by an expansion in a person’s huntingtin gene, which leads to an expanded huntingtin protein. For many years, scientists and clinicians understood that this caused neurodegeneration, but were uncertain as to how or why. On September 26, 1997, a group of scientists in Boston and London, including HDSA Scientific Advisory Board member Dr. Neil Aronin, published their findings that expanded huntingtin protein is resistant to removal from cells in the brain. The paper detailed that these expanded huntingtin proteins aggregate, and indicated that this phenomenon may play a role in neurodegeneration in HD. This was a groundbreaking discovery for how we understand the biological cause of HD symptoms.
