I think they have and that's why the approval is as it is. The selection process to be eligible for the trial also supports that fibrosis is a known problem. Thise boys, even if ambulant, are excluded from most trials if indications of a certain amount fibrosis damage to muscle is evident.
I sm an optimist, but this all looks very good fir ATL1102 as a mono therapy. Will be interesting to see if additional gene therapies add much additional benefit.
The last trial didnt take long to show improvement in PUL2 and against natural history. Drugs to market are allowed during approval trials if certain criteria are met. Any improvement for non ambulant boys could tick that box.
Drug in arms! Am still guessing second half June.
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