Open AccessLong-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial
Author(s) -
Laurent Servais,
Eugenio Mercuri,
Volker Straub,
Michela Guglieri,
Andrei Seferian,
Mariacristina Scoto,
Daniela Leone,
E. Koenig,
Navid Khan,
A. Dugar,
Xiaodong Wang,
Baoqin Han,
Dan Wang,
Francesco Muntoni,
Chiara Brusa,
Laura Antonaci,
Claudia Brogna,
Laura Merli,
Mauro Monforte,
Giulia Norcia,
Marika Pane,
Gloria Ferrantini,
George Dickson,
Jennifer E. Morgan,
Valentina Sardone,
Cody Akana,
Jay S. Charleston,
Cody A. Desjardins,
Saleh El-Husayni,
Diane E. Frank,
Frederick J. Schnell
Publication year - 2022
Publication title -
nucleic acid therapeutics
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.255
H-Index - 67
eISSN - 2159-3345
pISSN - 2159-3337
DOI - 10.1089/nat.2021.0043
Subject(s) - medicine , duchenne muscular dystrophy , clinical endpoint , adverse effect , ambulatory , population , clinical trial , placebo , phases of clinical research , dystrophin , pathology , alternative medicine , environmental health