Research Blitz: Neurocrine, Prilenia, uniQure, Wave, Novartis, Sage, and HDClarity

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  • Опубликовано: 5 сен 2024
  • (November 6, 2021) Dr. Ed Wild, of the University College, London shares on HDClarity, a multi-site initiative to collect and study cerebrospinal fluid (CSF) as a means of understanding the brain, collection initiative to facilitate therapeutic development for HD. HDClarity now has 25 sites globally.
    Dr. Aaron Koenig, MD from Sage Therapeutics talks about a future trial for HD, SAGE-718 focusing on the cognitive disorder in HD.
    Dr. Blair Leavitt, speaks on a new clinical HD lowering drug called Branaplam an oral htt lowering therapy for HD developed by Novartis, a global healthcare company, and VIBRANT-HD, A phase IIb study of Branaplam in HD.
    Dr. Michael Hayden speaks on the PROOF-HD study, exactly what is Pridopidine, the drug being given for this study, studying the daily functional capacity for the person with HD such as eating and dressing.
    This study is being conducted in 30 North American sites and 9 countries in Europe in collaboration with HSG, TFS, and CTCC.
    Dr. Ralf Reilmann, FAAN from the George Huntington Institut in Germany presents the Select-HD study conducted by WAVE Life Sciences. How preservation of wild-type HTT may help to support the health and function of the brain, distinct HTT-lowering approach, this is an adaptive first-in-human study for WVE-003. This study is moving forward globally.
    Dr. Erin Furr-Stimming is with the University of Texas Health Science Center in Houston, TX, and is presenting on the AMT-130 study conducted by uniQuire gene therapy one-time neurosurgical delivered intracranial injection into the most relevant brain region.
    Dr. Daniel Claassen from Vanderbilt University gives an update on the KINECT-HD study, a study designed to test the effectiveness of Valbenazine, conducted by Neurocrine Therapeutics for chorea associated with HD. The results of this trial will be known very soon.

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