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May 2025: This Month in Huntingtonās Disease Research
Latest News
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A first for CRISPR gene editing could have wider applications for human disease
A successful early trial of a drug for Familial Transthyretic Amyloidosis showed that CRISPR gene editing could be safely used in the human body. What does this mean for gene editing in HD?
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Scientists identify precisely how pridopidine works in models of Huntingtonās disease
New studies pinpoint precisely how the drug pridopidine works in models of Huntingtonās disease
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Huntington's disease therapeutics conference 2021 – Day 3
Catch up on all of the latest updates from day 3 of the 2021 CHDI Huntingtonās disease therapeutics conference #HDTC2021
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Huntingtonās disease therapeutics conference 2021 – Day 1
Catch up on all of the latest updates from day 1 of the 2021 CHDI Huntingtonās disease therapeutics conference #HDTC2021 including presentations from Wave, Roche and Triplet.
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Huntington's disease therapeutics conference 2021 – Day 2
Catch up on all of the latest updates from day 2 of the 2021 CHDI Huntingtonās disease therapeutics conference #HDTC2021
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Huntingtonās disease clinical trial round up
Catch up on all the latest Huntington's disease clinical trial news in this one stop shop article covering all of the recent developments in making medicines for Huntington's disease
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Good news from uniQure: gene therapy trial on track, and promising data in animals
The first group of 10 participants have been dosed in uniQureās clinical trial of an HD gene therapy, and three new manuscripts describe safe, widespread huntingtin lowering in animals.
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Disappointing Results from Waveās PRECISION-HD1 and 2 Trials
Wave Life Sciences shared the disappointing news that their two ASOs in Phase 1/2 trials in HD patients did not successfully lower mutant huntingtin.
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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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Vaccines and HD
Many HD families have questions about vaccination for the coronavirus – RNA sounds scary! HDBuzz helps unpack the headlines about vaccine safety and HD.