Nippon Shinyaku takes on Sarepta
A new competitor enters the exon 53 skipping niche, and might well take it over.
Go or no go? Valuable drugs set for FDA decisions
Upcoming US approval verdicts include inclisiran, valrox and risdiplam, but toxicity issues could scupper filgotinib.
Vertex’s second transformation: Duchenne time
The cystic fibrosis player does deals with Crispr and Exonics to launch itself into a highly competitive rare disease area.
