3 ms·
That's for one type of muscular dystrophy, SMA, there are many other types without great treatment options. Lifelong exon skipping treatments recently approved
by fullburley 5y ago
That's for one type of muscular dystrophy, SMA, there are many other types without great treatment options. Lifelong exon skipping treatments recently approved for subsets of DMD are >1M USD per year, and result in dystrophin expression at only a few percent of "normal" (see data from trials of casimersin).