There are known knowns and known unknowns. There are also unknown unknowns which makes it all the more difficult.
It looks like the stuff works in DMD nonambulant but there are many competing technologies after the same pot of gold including gene therapies which have the potential to cure the condition.
In spite of the unknowns the current knowns look very favourable to me hence recent top ups.
DYOR
GLLTH.
Cheers QS
Ann: Positive feedback received on Paediatric Investigation Plan, page-93
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