Neurological
A Two-Part, Open-Label Systemic Gene Delivery Study To Evaluate The Safety And Expression Of RO7494222 (SRP-9001) In Subjects Under The Age Of Four With Duchenne Muscular Dystrophy (ENVOL)
The purpose of this study is to assess the safety and expression of delandistrogene
moxeparvovec (also referred to as RO7494222 or SRP-9001), a novel micro-dystrophin gene
therapy, in participants under the age of 4 with Duchenne muscular dystrophy (DMD). Current
treatment options for DMD include systemic corticosteroids which confer limited benefit while
introducing significant side effects, and therapies aimed at restoring the production of
endogenous dystrophin protein (e.g., exon-skipping) which are only available to a small
proportion of patients with specific genetic mutations. Therefore, there continues ...
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