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 ...

TFC has been formally incorporated into the Unified Huntington’s Disease Rating Scale (UHDRS), which is the standard clinical ...

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 ...

INGREZZA is the trade name of valbenazine, a drug developed by the company Neurocrine Biosciences. It works similarly to tetrabenazine and deutetrabenazine (Austedo), drugs commonly prescribed to help ...

This month brought landmark therapeutic news, advances in biomarkers, deeper insights into what drives Huntington’s disease (HD), and fresh perspectives on mental health in HD. In particular among the ...

⏱️ 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 ...

In plain language. Written by scientists. For the global HD community.

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 ...