clinical-trial
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Skyhawk Shares A Closer Look at SKY-0515 Data, Plus A Peek At Expanding US Trial Access
⏱️ 6 min read | New 12-month data from Skyhawk’s SKY-0515 is in and all four measures that make up the cUHDRS are at or above baseline, beating expected natural decline. Plus, a first look at expanding US trial sites.
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The Other Shoe Drops: uniQure Shares Plans To Submit Licensing Application With The FDA for AMT-130
⏱️ 9 min read | After a turbulent past year, uniQure have shared encouraging news that the FDA has agreed their Phase I/II study data can serve as the basis for an application that could lead to the approval of AMT-130 in the US.
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First participant dosed in pioneering neural stem cell trial for Huntington’s disease
A new clinical trial for HD is underway. The first participant has been dosed in REGEN4HD, a study testing whether transplanting neural stem cells is safe as a potential treatment for HD.
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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.