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Additional Clarity: What We Know 4 Weeks After the uniQure News
Latest News
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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 uniQure’s 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 Wave’s 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.
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GPR52: Exploring a new way to lower huntingtin
A Chinese research team developed a new way to lower huntingtin protein indirectly, by targeting a protein called GPR52. The molecules they designed were protective in cells and in mice with HD.
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Uncovering the dark side of DNA repair to design HD treatments
A gene called MSH3 helps to repair our DNA, but in HD it can slip up and cause CAG repeats to lengthen. Researchers have uncovered new information about how MSH3 activity is controlled, opening the door to new therapeutic avenues.
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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 Huntington’s 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