Rare Disease Report

Huntingtons Disease

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.
George Yohrling, PhD discusses treatment for Huntington's disease patients, therapies in the pipeline and why it’s an exciting time for the research community.
Results from a recent study published in Lancet Neurology by researchers in England and Wales identifies a gene linked to Huntington’s disease progression.
In Huntington's disease animal models, AMT-130, an AAV5 vector carrying a DNA cassette encoding artificial micro-RNA, is effective.
Louise Vetter, CEO of the HDSA and George Yohrling, PhD discuss the creation of the organization, what motivates them, and why their work is so important.
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