On April 30, 2026, uniQure announced that it held a Pre-Submission Meeting with the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) and plans to file a formal application ...
On May 5th, PTC Therapeutics released results from their ongoing Phase 2 PIVOT-HD clinical trial for PTC-518, now called votoplam. Excitingly, they announced that this trial met its primary endpoint – ...
10 min read | PTC Therapeutics shared 2-year data for votoplam, a daily HTT-lowering pill. Stage 2 participants showed up to ...
8 min read | April 2026 showed us a toxic HTT fragment is center stage in mouse research, CRISPR is showing promise in mouse ...
Long repetitive sequences of C-A-G letters in the DNA code are associated with at least 12 genetic diseases, including Huntington’s disease (HD). A group of scientists in Massachusetts, USA, have ...
Apathy in people with Huntington’s disease (HD) isn’t just a lack of interest or motivation, it is a loss of drive, initiative, and emotional responsiveness that affects not only what the person with ...
The Total Functional Capacity scale, or TFC, was developed in 1979 by researchers Dr. Stanley Fahn and the late Dr. Ira Shoulson, founder of the Huntington Study Group. They wanted to describe the ...
Huntington’s disease (HD) is caused by a repetition of the genetic letters C-A-G in the huntingtin gene. People who won’t develop HD have 35 or fewer CAGs, whereas people who go on to develop HD have ...
HDBuzz has always been free to read, and we intend to keep it that way. But free to read doesn’t mean free to run. As an independent nonprofit powered entirely by our community, we’re launching Fund ...
In plain language. Written by scientists. ⏱️ 10 min read | The Total Functional Capacity score has been used in Huntington’s disease research for decades. Here’s what it measures, what it misses, & ...
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