First Gene-Silencing Drug Approved by the FDA

Posted on August 16, 2018

This week marked the first FDA approval of a gene silencing drug, for a rare hereditary disease called ATTR amyloidosis. A successful trial determined that the drug, ONPATTRO (patisiran), helped with people’s symptoms, like polyneuropathy (pain from nerve damage), dizziness, and stomach issues, and improved quality of life, activities of daily living, and nutritional status….

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Roche Huntingtin-Lowering Drug Gains PRIME Status from EMA

Posted on August 7, 2018

On August 4th, Roche Pharmaceuticals announced that their huntingtin-lowering therapy was granted PRIME status from the European Medicines Agency (EMA). The EMA is the European equivalent of the United States Food and Drug Administration (FDA). In Europe, PRIME (PRIority MEdicines) designation means that the EMA has agreed that this drug, RG6042 (formerly IONIS-HTTRx) could potentially…

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Voice Your Opinions About The Future Of CRISPR

Posted on August 2, 2018

Community research surveys are valuable ways for researchers to get input from HD patients, families and caregivers about a variety of topics, and can help guide strategies for HD research and care. HDSA works with scientists and students to make their university-approved research surveys available for easy online participation through our website. This week, there’s…

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At-Home DNA Kits Require Caution

Posted on July 26, 2018

At-home DNA kits like 23 & Me are growing in popularity, and claim to provide information about your health and ancestry. The Washington Post created an FAQ about what you can actually expect to learn – and who gets your info. These kits do not test for genetic disorders like Huntington’s disease. For more information…

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Pipeline of Therapies for HD

Posted on July 19, 2018

The Rare Disease Report covers stories about medicine, finance, and advocacy in rare disease fields. Last week an article explored some of the promising therapies in the pipeline for HD. To learn more about advances in HD research, check out HDSA’s page detailing current preclinical and clinical research studies and their current phase of development….

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DNA Repair Research Uncovers New Drug Targets

Posted on July 12, 2018

Researchers led by Ray Truant at McMaster University in Canada are investigating how DNA repair could help correct some of the damage caused by mutant huntingtin. They tested a molecule that is processed by the body to signal that DNA repair needs to take place. Giving mice high levels of this molecule helped to improve…

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Research Highlights from the 33rd Annual HDSA Convention

Posted on July 5, 2018

As we ease into the summer, enjoy some highlights from our research sessions at HDSA’s 33rd Annual Convention in Los Angeles. The Ionis/Roche ASO drug has gotten lots of press in the past year, but did you know there are additional strategies for lowering huntingtin? Multiple companies and academic laboratories are approaching this goal from…

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HDSA’s 33rd Annual Convention Research Forum Video Now Available

Posted on June 14, 2018

HDSA’s 33rd Annual Convention in Los Angeles from June 7th-9th included the 2018 Research Forum, where ten speakers collaborated to tell a story about the HD gene and the continued work towards designing and testing a huntingtin-lowering therapy. Check it out here and stay tuned for a recap of research news at Convention!   CRISPR research…

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HDSA Convention Research Forum

Posted on June 7, 2018

This week we’re hosting the 33rd Annual HDSA Convention in Los Angeles, California. Tune in to the livestream of our Research Forum this Saturday, June 9th beginning at 9 am PDT (12 pm EDT). Sessions will also be recorded for later viewing. Visit for more info and follow us on social media with #HDSAConvention.

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Huntington’s Disease Featured in Nature Outlook

Posted on May 31, 2018

The prestigious journal Nature publishes both research reports and journalistic articles, and is widely read by scientists and non-scientists. This week, Nature published an Outlook edition specifically related to Huntington’s disease. The articles explore how scientists are approaching the biology of HD and Juvenile HD, invite perspectives from at-risk and affected individuals, discuss huntingtin-lowering therapies…

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