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Safety, tolerability and pharmacokinetics of eteplirsen in young boys aged 6-48 months with Duchenne muscular dystrophy amenable to exon 51 skipping.
Mercuri E, Seferian AM, Servais L, Deconinck N, Stevenson H, Ni X, Zhang W, East L, Yonren S, Muntoni F; 4658-102 Study Group. Mercuri E, et al. Among authors: east l. Neuromuscul Disord. 2023 Jun;33(6):476-483. doi: 10.1016/j.nmd.2023.03.008. Epub 2023 Mar 24. Neuromuscul Disord. 2023. PMID: 37207382 Free article.
Corrigendum to 'Population pharmacokinetics-based recommendations for a single delayed or missed dose of nusinersen': Neuromuscular Disorders 31 (2021) 310-318/doi: 10.1016/j.nmd.2021.02.014.
MacCannell D, Berger Z, East L, Mercuri E, Kirschner J, Muntoni F, Farrar MA, Peng J, Zhou J, Nestorov I, Farwell W, Finkel RS. MacCannell D, et al. Among authors: east l. Neuromuscul Disord. 2022 Jan;32(1):e1. doi: 10.1016/j.nmd.2022.01.002. Neuromuscul Disord. 2022. PMID: 35144755 Free article. No abstract available.
Safety, tolerability, and pharmacokinetics of casimersen in patients with Duchenne muscular dystrophy amenable to exon 45 skipping: A randomized, double-blind, placebo-controlled, dose-titration trial.
Wagner KR, Kuntz NL, Koenig E, East L, Upadhyay S, Han B, Shieh PB. Wagner KR, et al. Among authors: east l. Muscle Nerve. 2021 Sep;64(3):285-292. doi: 10.1002/mus.27347. Epub 2021 Jun 29. Muscle Nerve. 2021. PMID: 34105177 Free PMC article. Clinical Trial.
122 results