Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy.

Diane E FrankFrederick J SchnellCody AkanaSaleh H El-HusayniCody A DesjardinsJennifer MorganJay S CharlestonValentina SardoneJoana DomingosGeorge DicksonVolker StraubMichela GuglieriEugenio MercuriLaurent ServaisFrancesco Muntoninull null

Published in: Neurology (2020)

This study provides Class I evidence that golodirsen is safe and Class IV evidence that it induces exon skipping and novel dystrophin as confirmed by 3 different assays.

Keyphrases

duchenne muscular dystrophy
muscular dystrophy
high throughput