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 – ...
In plain language. Written by scientists. ⏱️ 6 min read | While the US regulatory path for AMT-130 remains complicated, uniQure announced plans to seek approval for its HD gene therapy in the UK. Here ...
10 min read | PTC Therapeutics shared 2-year data for votoplam, a daily HTT-lowering pill. Stage 2 participants showed up to ...
Jenni Nguyen is a Cell and Developmental Biology PhD student at the University of British Columbia. After completing her B.A. in Biological Sciences at Wellesley College, she spent a few years working ...
Two articles this month took aim at the root cause of HD rather than its downstream consequences. The HTT1a mouse study and the CRISPR study are asking the same fundamental question from different ...
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 ...
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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