clinical-trial
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Skyhawk reports encouraging 12-month results for oral Huntington’s disease drug SKY-0515
One year into treatment, SKY-0515 continues to lower huntingtin and shows encouraging trends with signs and symptoms of HD. A larger trial is enrolling to determine whether the drug truly slows HD progression
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A Second Path: uniQure Plans Regulatory Filing for AMT-130 in the UK
⏱️ 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’s what that means, and what might be to come.
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Two Years In: New Long-Term Extension Data from PIVOT-HD for Votoplam
⏱️10 min read | PTC Therapeutics shared 2-year data for votoplam, a daily HTT-lowering pill. Stage 2 participants showed up to 52% slowing of disease progression. Here’s what the data show, what’s still missing, and the roll out of the Phase 3 trial.
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The Number on the Scale: What the TFC Score Measures, and Why It Matters Right Now
⏱️ 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, & why it’s at the center of an anticipated clinical trial.
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Your Wrist on Watch: Could a Smartwatch Reveal Huntington’s Disease Symptoms?
⏱️ 7 min read | A wrist sensor tracked arm movements in people with HD for a week and could see who had HD and who didn’t. This kind of technology could change how we measure drug effects in trials.
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Australia Opens The Door for SKY-0515: Skyhawk Seeks Provisional Approval for Its Oral HD Drug
⏱️ 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 pill to people with HD sooner.
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The road ahead for uniQure: FDA says more data needed for AMT-130 gene therapy
⏱️ 10 min read | The FDA wants more data before approving AMT-130 for Huntington’s disease in the U.S. On March 2026, uniQure shared in an update that current Phase 1/2 data weren’t enough for the agency. A new randomized, sham-controlled trial may be required.
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Vico’s Trial Adjusts With Twice-Yearly Dosing, And The U.S. Is Next
⏱️ 6 min read | Vico Therapeutics’ Huntington’s disease drug VO659 is now being tested just twice a year, and the FDA has cleared the way for US trials to begin later this year.
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Putting it in print: oral drug branaplam lowered huntingtin, but safety concerns halted development
⏱️10 min read | Results from the VIBRANT-HD trial are now published in a peer reviewed journal. This trial tested the oral drug, branaplam, which lowered HTT but had serious safety problems, ultimately halting the trial.
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Phase 1 results announced for Skyhawk’s drug SKY-0515
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 is an encouraging step as larger trials continue.