WebOct 23, 2024 · Branaplam is giving hope in Huntington's disease. (Taljat David/Shutterstock) After discovering promising indicators on the path to the development of Branaplam (LMIO70) for spinal muscular atrophy (SMA), Novartis now hopes to repurpose the drug for the treatment of Hungtington’s disease. The U.S. Food and Drug Administration (FDA) … WebMar 4, 2024 · As experimental treatments for Huntington’s disease continue to suffer setbacks, Novartis is outlining a plan for a repurposed SMA drug it hopes can break pharma’s losing streak.
Novartis to pursue SMA drug branaplam in Huntington
WebMar 21, 2024 · Huntington’s disease is a devastating neurodegenerative disease caused by a CAG repeat in the first exon of the huntingtin gene. This mutation causes brain cells to die, leading to a myriad of progressive cognitive, psychiatric and movement disorders. WebFeb 2, 2024 · Branaplam/Novartis. Huntington’s disease. ... Huntington’s disease. 10/19/2024 FDA put a clinical hold on phase 2 trial for this small-molecule splicing modulator, requesting more evidence. sign into bmo rewards
Novartis Discontinues Huntington Disease Program Following …
WebFeb 1, 2024 · Novartis Discontinues Huntington Disease Program Following Phase 2b Study Feb 1, 2024 Isabella Ciccone, MPH Branaplam, an mRNA splicing modifier, joins the many unsuccessful agents for Huntington disease as biopharma continues to be challenged with trial holds and failures. WebDec 13, 2024 · Huntington’s is caused by mutations in the gene HTT, which provides instructions to make the huntingtin protein. Disease-causing mutations result in the production of an abnormally long version of this protein, which forms toxic clumps in nerve cells. Reducing levels of this toxic protein is widely seen as a potential treatment strategy. 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. sign in to blink