RE:Big Pharma walks away from gene-therapy biotech May 10, 2023 - Is there enough evidence to support accelerated approval for use in ambulatory patients who have a confirmed mutation of the DMD gene?
The FDA, for its part, seems to be unconvinced.
https://www.fiercebiotech.com/biotech/fda-questions-benefits-risks-sarepta-dmd-gene-therapy-concern-thats-thrived-2018