disease-modifying
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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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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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Additional Clarity: What We Know 4 Weeks After the uniQure News
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 from the Huntington’s disease community?
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Blue skies for Skyhawk: Positive news from Phase 1 trial for SKY-0515
There’s more good news in the forecast in the Huntington’s disease therapeutic space as we receive positive results from Skyhawk Therapeutics about their small molecule SKY-0515 that lowers huntingtin and targets somatic expansion.
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Hereditary Disease Foundation (HDF) conference 2022 – Day 1
Read updates from clinical trials and scientific research on Huntington’s disease from Day 1 of the 2022 HDF Milton Wexler Biennial Symposium #HDF2022
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Serious side effects reported for some people treated with the huntingtin-lowering drug AMT-130, currently in clinical trials
After receiving a high dose of uniQure’s gene therapy for Huntington’s disease, a few patients experienced serious side effects, but are now recovering. HDBuzz explores what this means for the experimental huntingtin-lowering drug, AMT-130.
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Sad News from Roche and Ionis – ASO Trial Halted Early
Disappointing news from Roche and Ionis; the phase III Tominersen huntingtin-lowering trial has been halted early
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Huntington's disease goes viral as UniQure inches ahead in gene therapy race
FDA grants “Investigational New Drug” status to Huntingtin-lowering gene-therapy agent AMT-130, clearing path to human trials in Huntington’s Disease patients
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Advances on many fronts in the battle against the protein that causes Huntington's disease
This fall sees exciting announcements from a number of companies focused on novel Huntingtin Lowering technologies, including Wave, PTC and Voyager