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Vamorolone for treating Duchenne muscular dystrophy in people 4 years and over (TA1031)
Evidence-based recommendations on vamorolone (Agamree) for treating Duchenne muscular dystrophy in people 4 years and over.
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Evidence-based recommendations on ataluren (Translarna) for Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene in people 2 years and over who can walk.