clinical-trial
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AMT-130 at Four Years: Hopeful Signs, Complex Controls
12 min read
uniQure reports that after 4 years, people given high-dose AMT-130 continue to show signs of slower HD progression, providing hope for their recent regulatory submission. But the new analyses are nuanced. We unpack the hopeful, but complex results.
By Dr Leora Fox -
Encouraging news from Skyhawk, as SKY-0515 Phase 1/2 data continues to hold promise
10 min read
Skyhawk shared data at the 15 month mark for SKY-0515 treatment in people with HD in their Phase 1/2 trial. The drug is working as designed and there are some encouraging signs that it could be helpful for some symptoms of HD.
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First participant dosed in the PRECISE-HD trial of pridopidine
4 min read
The first participant has now been dosed in the Phase 3 PRECISE-HD trial of pridopidine for HD. The study will test whether the drug can slow disease progression in people most likely to benefit
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A shot in the arm for HTT lowering: INSIGHTT trial begins testing SRP-1005
6 min read
A new Phase 1 study is testing SRP-1005, a huntingtin-lowering therapy given by injection under the skin. It’s early days, but this approach could add an important new arrow to the HD drug development quiver.
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Prilenia shares HD trial data with CHDI to help future research
6 min read
Clinical trial data can drive discovery long after a study ends. Prilenia donated placebo-arm data from 2 HD clinical trials to CHDI, a valuable resource for researchers to better understand placebo effects, HD progression and future trial design.
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Looking back and moving forward: Questions and lessons 4 weeks out from Roche’s disappointing news
10 min read
Roche’s decision to stop development of 2 HTT-lowering programmes is disappointing, but the story doesn’t end there. Years of research answered some key questions, raised many new ones, and will inform the next generation of HD trials.
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Putting it in print: a clinical trial trying to rein in runaway pruning
9 min read
Four years after announcing positive results in their trial of ANX005 (tanruprubart) for HD, Annexon has formally published the results. It’s safe, hits its target, and may have helped some participants – so what’s the holdup?
By Dr Leora Fox -
Roche ends two Huntington’s disease drug programmes after disappointing results
10 min read
Roche announced it is stopping development of two of its huntingtin-lowering ASO drugs, tominersen and RG6496. This marks a disappointing end to an important chapter in HD clinical research.
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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
8 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.