Re: First commercial patient treated with Elevidys
Exondys: no clinical efficacy shown yet. In fact, the label clearly indicates a *lack* of clinical efficacy (by the 6MWT data in the submission package). Confirmatory trial ongoing, no data yet. All published literature points to this lack of (proof of) efficacy.
Vyondys: they didn't even try to show clinical efficacy in their filing package. Microdystrophin only.
All published literature (check pubmed) points to the efficacy of this whole approach being very much "an open question".
The above are simply facts, not opinions.