First commercial patient treated with Elevidys | SRPT Message Board Posts


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Msg  14608 of 14657  at  8/3/2023 7:48:03 PM  by

Pritu


 In response to msg 14605 by  redplate
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Re: First commercial patient treated with Elevidys

Really.

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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