huntingtin-lowering
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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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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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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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What does COVID-19 mean for Huntington’s 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?
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Huntington’s disease therapeutics conference 2020 – Day 3
HDBuzz reports from the annual Huntington’s disease therapeutics conference in Palm Springs
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Unpacking Wave's PRECISION-HD2 huntingtin-lowering trial announcement
Wave Life Sciences announces that its antisense drug WVE-120102 has lowered mutant huntingtin protein in cerebrospinal fluid, but investors seem disappointed. Rather confusing – what do we know for sure?
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Could molecular handcuffs lower the protein that causes Huntington's disease?
Researchers got surprisingly lucky when looking for drug molecules to pull mutant huntingtin protein into a cellular garbage disposal machine
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Exciting new Huntingtin lowering tool described
Exciting new Huntingtin Lowering work from @SangamoTx and @CHDIfoundation using "Zinc Fingers" to shut down expression of the mutant Huntingtin gene. More details on this exciting new technique here.
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Details emerge of first Huntington’s disease gene therapy clinical trial
UniQure announces key details of its planned trial to assess the safety and ability of AMT-130 gene therapy to lower the problematic huntingtin protein using a ‘single-shot’ virus delivery system.