Rare Disease Report

Huntingtons Disease

The study of IONIS-HTTRx was completed. The Phase 1/2a evaluated the first therapy in clinical development intended to target the underlying cause of Huntington’s Disease (HD).
RDR spoke with George Yohrling of the HDSA about AMT-130, uniQure N.V.’s gene therapy which received an Orphan Drug Designation in October.
With altering the immune system in HD patients emerging as such a hot topic, Rare Disease Report sat down with George Yohrling to get his thoughts on whether this method is worth pursuing.
A new study revealed that ATF5 proteins can become implanted in other substances in the brains of patients with Huntington’s disease.
Is the immune system involved in the progression of HD? And can drugs that target the immune system help HD patients?
Can rilmenidine delay disease progression in Huntington's disease? Cambridge researchers are trying to find out.
Louise Vetter discusses 50 years of the HDSA, and insight as to why she continues to fight on behalf of families affected by Huntington's disease.
The administration of AMT-130 into the mouse striatum led to significant improvements in motor symptoms and survival.
Louise Vetter stresses the importance of storytelling in rare diseases, and why film is such a powerful tool to convey messages that need to be heard.
In this video, Louise Vetter, President and CEO of the HDSA talks about Marjorie Guthrie’s impact on the rare disease community and the creation of NORD.
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