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 ...
We’re 4 weeks out from uniQure’s positive update about AMT-130 that took the world by storm. Does the data match the media hype? Where do uncertainties remain? And what are some of the key questions ...
⏱️ 8 min read | An oral Huntington’s disease drug is eligible for accelerated approval in Australia. This isn’t full approval yet, but it opens a faster path to potentially getting this once-daily ...
Skyhawk Therapeutics shared results for SKY-0515, a drug designed to lower huntingtin. The drug appeared generally safe, with 60% reduction of huntingtin in blood at the highest dose tested. This data ...
This week, we heard an update from Roche about their huntingtin-lowering therapy, tominersen, currently being tested in the GENERATION HD2 trial. An independent data monitoring committee (iDMC) that ...
For those who were following the live tweets from HDBuzz about the CHDI HD Therapeutics Conference or tuned in to the HDSA Convention, we may have caught your attention with the new HD staging system.
The SIGNAL clinical trial was designed to test a drug called pepinemab in people with early Huntington’s disease. The key results of that trial were recently announced, and unfortunately, pepinemab ...
A 14-year study tracking NfL levels in people with the HD gene shows this tiny protein can signal disease progression many years before symptoms start. The longest follow-up study yet adds weight to ...
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