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Re: First commercial patient treated with ElevidysReally. 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. Amondys: same. 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. |
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