Rare Disease Report

Huntingtons Disease

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.
Intracerebroventricular infusion of GM1 resulted in numerous improvements in neuropathology, behavior, and cognition in HD mice models
AMT-130 has a AAV5 vector carrying an artificial micro-RNA designed to silence the mutant huntingtin gene.
A new study recently published in JAMA Neurology may have found a solution to the factors limiting HD clinical trials.
HDWear, a technology that utilizes sensors to monitor motor function of Huntington’s disease patients, has received more than $2.5 million from the NIH.
Three global Huntington’s disease patient advocacy organizations have teamed up to give those affected by the disease a voice in clinical research.
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