Quay,
"...including gene therapies which have the potential to cure the condition."
Gene Therapy ain't curing DMD anytime soon.
Read the Sarepta and Pfizer releases from last couple of years to get the full picture...or the analyst reports on Antisense website.
Safety concerns...and lack of efficacy are big issues.
FDA has placed a hold on Pfizer's GT program in the US due to safety issues.
Treatments that are pursuing additional dystrophin are not showing efficacy.
Not many sleeps until EMA approval.
.
Ann: Positive feedback received on Paediatric Investigation Plan, page-94
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