Ann: WHO selects proposed INN for ATL1102, page-11

https://www.biospace.com/article/as-sarepta-s-elevidys-closes-in-on-full-approval-experts-weigh-in

With the deaths of young DMD boys and the long term side effects in question.. I can’t see the FDA passing the gene therapy drug .
Not only that the high cost and the drug failed to show efficacy
Imo
the FDA will not give approval for the entire DMD population

PER is only days away from full enrolment ..
Then the countdown begins…
We already have more than 10 boys who are dosing beyond the 6 month period
All good.
Also the Tox study is completed or near enough
the fact that they haven’t stoped the trial tells me that the 9 month study is successful.

..