Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI Trial.
McDonald CM, Shieh PB, Abdel-Hamid HZ, Connolly AM, Ciafaloni E, Wagner KR, Goemans N, Mercuri E, Khan N, Koenig E, Malhotra J, Zhang W, Han B, Mendell JR; the Italian DMD Telethon Registry Study Group, Leuven NMRC Registry Investigators, CINRG Duchenne Natural History Investigators, and PROMOVI Trial Clinical Investigators.
McDonald CM, et al. Among authors: zhang w.
J Neuromuscul Dis. 2021;8(6):989-1001. doi: 10.3233/JND-210643.
J Neuromuscul Dis. 2021.
PMID: 34120909
Free PMC article.
Clinical Trial.