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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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Fresh Updates from First Huntingtin Lowering Study Publication
Hot off the presses – New publication gives more details about the results of Ionis and Roche's safety study with a Huntingtin-lowering ASO
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When interrupting is good: genetic hiccups that protect against Huntington's disease
Multiple teams find small differences in the 'CAG repeat' bit of the Huntington's disease gene. They don't directly change the huntingtin protein, but do alter the age of symptom onset. What's behind this enigma and what does it mean for patients?
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Huntington's disease therapeutics conference 2019 – Day 1
HDBuzz reports from the annual Huntington’s disease therapeutics conference in Palm Springs
By Joel Stanton -
HDSA FAQ on the Roche/Genentech RG6042 program
HDSA's frequently asked questions factsheet about the huntingtin-lowering trial ASO program was so good we stole it (with permission)
By Dr Leora Fox -
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
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Switch off the genome editor when you’re done
CRISPR genome editing just got an off-switch. We cut through the hype to explore the technology in HD
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Update confirms Huntington's disease 'gene silencing' trial on track
Ionis says its trial of HTTRx, intended to lower huntingtin protein, is fully recruited and plans to extend it
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FDA approves a new drug for symptoms of Huntington's disease
Deutetrabenazine becomes the first new drug in a decade licensed by the FDA for symptoms of Huntington's disease
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Pfizer Amaryllis trial ends in disappointment: no improvement in Huntington's disease symptoms
Pfizer announces the 'Amaryllis' trial of a PDE-10 inhibitor drug failed to improve symptoms of HD