clinical-trial
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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
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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 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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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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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.
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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
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Disappointing news from LEGATO-HD trial of laquinimod in Huntington's disease
The LEGATO-HD trial of laquinimod did not slow progression of Huntington's disease. Here's the lowdown.
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Success! ASO drug reduces levels of mutant protein in Huntington's disease patients
Amazing news from Ionis and Roche! HTTRx drug successfully lowers harmful huntingtin protein in spinal fluid