Sarepta FDA Hearing, page-42

Perhaps “cure” was too strong a word. In its optimal efficacy offering, my understanding is that SRP9001 is aiming to replace lack of dystrophin with micro-dystrophin in a sub-group of DMD patients, thereby stemming muscular dystrophy. Its efficacy is not the point (although I thought the ENDEAVOR study showed improvements in NSAA values). The point is it offers HOPE of stemming the degenerative effects of the disease before most of the these negative effects have started. With FDA support and full reimbursement, this “hope” treatment costs the parents nothing.

ANP chose to treat non-ambulant boys in trials (due to the increased prevalence of the target receptor CD49b at this stage of the disease). Although the recent mdx combo study aims in a new direction, development to commercial success of ATL1102 through the current P2b trial and beyond will likely (as I understand) eventuate in approval to treat non-ambulant DMD sufferers. The treatment does not aim to substitute or replace dystrophin, but stem the degeneration of muscle function by limiting inflammation. Not telling anyone anything they don’t know.

The oncology analogy is not perfect, but illustrates my point about arguable different motivations at different disease stages.

It might explain to some extent why ANPs treatment is not apparently subject to much DMD parent attention. The previous non-US focus also probably had something to do with it.

Just an opinion.