|
|
|
|
|
|
by fullburley
1758 days 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). |
|
|