WebDec 16, 2024 · Dec 16, 2024. Novartis today announced that the US Food and Drug Administration (FDA) has granted Fast Track designation for branaplam (LMI070) for the … WebHuntington’s disease. Novartis has received orphan drug status for branaplam to be used in a clinical trial to treat people with Huntington’s disease, which should launch this year. PTC Therapeutics also has a splice modulator called PTC518 which reduces the levels of huntingtin in different animal and lab models of Huntington’s disease.
First small-molecule drug targeting RNA gains momentum
WebMar 3, 2024 · Huntington's Disease (HD) is a progressive neurodegenerative disorder caused by CAG trinucleotide repeat expansions in exon 1 of the huntingtin (HTT) gene. The … WebFeb 1, 2024 · Courtesy of Getty Images. Novartis is cleaning house, cutting its Huntington’s disease program along with several others. The company announced multiple program stops and delays in its full-year 2024 report Wednesday.. August brought a temporary halt to the Phase IIb trial of branaplam for Huntington’s after the discovery of possible side … blinky x oc fanfiction
HDSA Statement Regarding VIBRANT-HD Trial - Huntington
WebFeb 8, 2024 · Tina Leggett, on behalf of the European Huntington Association, travelled to the Novartis Campus in Switzerland. She interviewed Dimitri Papanicolaou, Jang H... WebApr 6, 2024 · This Week In Huntington's Disease Research keeps you up-to-date on HDSA research activities, recently published work about Huntington’s disease, historical moments in HD research and more. HD Buzz on the Brain, Vico begins dosing, New Podcast Posted on April 6, 2024 How does an HD brain find balance? WebOct 21, 2024 · ZURICH (Reuters) - Novartis is seeking to repurpose its investigational oral spinal muscular atrophy (SMA) drug branaplam to treat Huntington's disease, the Swiss drugmaker said on... fred\u0027s custom tackle chilliwack bc