Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy Article Swipe
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Diane E. Frank
,
Frederick J. Schnell
,
Cody Akana
,
Saleh El-Husayni
,
Cody A. Desjardins
,
Jennifer E. Morgan
,
Jay S. Charleston
,
Valentina Sardone
,
Joana Domingos
,
George Dickson
,
Volker Straub
,
Michela Guglieri
,
Eugenio Mercuri
,
Laurent Servais
,
Francesco Muntoni
,
on behalf of the SKIP-NMD Study Group
·
YOU?
·
· 2020
· Open Access
·
· DOI: https://doi.org/10.1212/wnl.0000000000009233
· OA: W3010225431
YOU?
·
· 2020
· Open Access
·
· DOI: https://doi.org/10.1212/wnl.0000000000009233
· OA: W3010225431
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.
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