clinical-trial
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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.
By Dr Leora Fox -
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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Huntington Study Group (HSG) 2020 Annual Conference: HD in Focus – Day 2
The 2020 virtual HSG conference HD in Focus continues on Day 2 with a focus on clinical trials and drugs in development.
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Huntington Study Group (HSG) 2020 Annual Conference: HD in Focus – Day 1
Read our breakdown of some of the talks and presentations at day 1 of the Huntington Study Group (HSG) 2020 annual conference: HD in focus
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Treatment for neurological disorder could be repurposed for Huntingtonās disease patients
While developing a drug called branaplam for patients with SMA, the pharmaceutical company Novartis discovered that it could hold promise for people with HD. The FDA has granted a special status called Orphan Drug Designation.
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Updates from the EHDN Plenary Meeting 2020
Read our summary of the latest updates from the EHDN Plenary Meeting 2020
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Sad news from the SIGNAL study: pepinemab does not influence HD symptoms
The SIGNAL study did not meet its key clinical goals for #HuntingtonsDisease to slow or improve HD symptoms, but the results are still informative for the HD community and other fields.
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HD Young Adult Study defines the sweet spot: symptom-free with measurable changes
We know that HD-related changes can occur many years before symptom onset, but how early do those changes begin? A team of researchers set out to determine that with a new comprehensive study in pre-manifest HD young adults.
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What does COVID-19 mean for Huntingtonās disease families and HD research?
COVID-19 update: what does it mean for HD families, how does it impact HD research, and how has it changed the way science works?