Huntington’s disease is a rare, inherited neurodegenerative disease that leads to progressive disability and death
There are no approved disease modifying therapies that delay disease onset or slow progression of the disease
Branaplam (LMI070) is an orally administered, small molecule RNA splicing modulator that could potentially reduce the levels of mutant huntingtin protein
http://www.pharmastrategies.org/wp-content/uploads/2019/12/Pharma-Strategies-Website-Logo-300x138.png00English RSS Feedhttp://www.pharmastrategies.org/wp-content/uploads/2019/12/Pharma-Strategies-Website-Logo-300x138.pngEnglish RSS Feed2020-10-21 06:15:002020-10-21 06:15:00Novartis receives US Food and Drug Administration (FDA) Orphan Drug Designation for branaplam (LMI070) in Huntington’s disease (HD)
