Letter to the Editor: In response to P.R. Clemens et al., Efficacy and Safety of Viltolarsen in Boys with Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study, and Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy.
Oligonucleotides; viltolarsen; Humans; Male; Clinical Trials, Phase II as Topic; Muscular Dystrophy, Duchenne; Men; Neurology; Neurology (clinical)
Disciplines :
Pediatrics
Author, co-author :
Muntoni, Francesco; Dubowitz Neuromuscular Centre and MRC Centre for Neuromuscular Diseases, UCL Great Ormond Street Institute of Child Health & Great Ormond Street Hospital for Children Foundation Trust, London, UK
Straub, Volker; Institute of Genetic Medicine, University of Newcastle upon Tyne, Newcastle upon Tyne, UK
Servais, Laurent ; Université de Liège - ULiège > Département des sciences cliniques ; Department of Pediatrics, University of Oxford, Oxford, UK
Letter to the Editor: In response to P.R. Clemens et al., Efficacy and Safety of Viltolarsen in Boys with Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study, and Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy.
Clemens PR, Rao VK, Connolly AM, Harper AD, Mah JK, McDonald CM, Smith EC, Zaidman CM, Nakagawa T; CINRG DNHS Investigators; Hoffman EP. Efficacy and Safety of Viltolarsen in Boys With Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study. J Neuromuscul Dis. 2023;10(3):439-47.
Clemens PR, Rao VK, Connolly AM, Harper AD, Mah JK, McDonald CM, Smith EC, Zaidman CM, Nakagawa T; CINRG DNHS Investigators; Hoffman EP. Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy. J Neuromuscul Dis. 2022;9(4):493-501.
Clemens PR, Rao VK, Connolly AM, Harper AD, Mah JK, Smith EC, McDonald CM, Zaidman CM, Morgenroth LP, Osaki H, Satou Y, Yamashita T, Hoffman EP; CINRG DNHS Investigators. Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A Phase 2 Randomized Clinical Trial. JAMA Neurol. 2020;77(8):982-91.
Goemans N, Signorovitch J, Sajeev G, Yao Z, Gordish-Dressman H, McDonald CM, Vandenborne K, Miller D, Ward SJ, Mercuri E; investigators from PRO-DMD-01 Study, CINRG DNHS, ImagingDMD, and the DMD Italian Group. Suitability of external controls for drug evaluation in Duchenne muscular dystrophy. Neurology. 2020;95(10):e1381-e1391.
Goemans N, Vanden Hauwe M, Signorovitch J, Swallow E, Song J; Collaborative Trajectory Analysis Project (cTAP). Individualized Prediction of Changes in 6-Minute Walk Distance for Patients with Duchenne Muscular Dystrophy. PLoS One. 2016;11(10):e0164684.
Muntoni F, Domingos J, Manzur AY, Mayhew A, Guglieri M; UK NorthStar Network; Sajeev G, Signorovitch J, Ward SJ. Categorising trajectories and individual item changes of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy. PLoS One. 2019;14(9):e0221097.
Muntoni F, Signorovitch J, Sajeev G, Lane H, Jenkins M, Dieye I, Ward SJ, McDonald C, Goemans N, Niks EH, Wong B, Servais L, Straub V, Guglieri M, de Groot IJM, Chesshyre M, Tian C, Manzur AY, Mercuri E, Aartsma-Rus A; Association Française Contre Les Myopathies; on behalf of Universitaire Ziekenhuizen Leuven Group, PRO-DMD-01, The UK NorthStar Clinical Network, CCHMC, and The DMD Italian Group. DMD Genotypes and Motor Function in Duchenne Muscular Dystrophy: A Multi-institution Meta-analysis With Implications for Clinical Trials. Neurology. 2023;100(15):e1540-e1554.
Mercuri E, Signorovitch JE, Swallow E, Song J, Ward SJ; DMD Italian Group; collaborative Trajectory Analysis Project (cTAP).Neuromuscul Disord. 2017;27(5):e1.
Categorizing natural history trajectories of ambulatory function measured by the 6-minute walk distance in patients with Duchenne muscular dystrophy” [Neuromuscular Disorders 26/9 (2016) 576-583].
Servais L, Mercuri E, Straub V, Guglieri M, Seferian AM, Scoto M, Leone D, Koenig E, Khan N, Dugar A, Wang X, Han B, Wang D, Muntoni F; SKIP-NMD Study Group. Long-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. Nucleic Acid Ther. 2022;32(1):29-39.
Aartsma-Rus A, Morgan J, Lonkar P, Neubert H, Owens J, Binks M, Montolio M, Phadke R, Datson N, Van Deutekom J, Morris GE, Rao VA, Hoffman EP, Muntoni F, Arechavala-Gomeza V; workshop participants. Report of a TREAT-NMD/World Duchenne Organisation Meeting on Dystrophin Quantification Methodology. J Neuromuscul Dis. 2019;6(1):147-59. doi: 10.3233/JND-180357
Frank DE, Schnell FJ, Akana C, El-Husayni SH, Desjardins CA, Morgan J, Charleston JS, Sardone V, Domingos J, Dickson G, Straub V, Guglieri M, Mercuri E, Servais L, Muntoni F; SKIP-NMD Study Group. Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy. Neurology. 2020;94(21):e2270-e228
Reply to F. Muntoni et al.: “In response to P.R. Clemens et al, Efficacy and Safety of Viltolarsen in Boys with Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study, and Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy. J Neuromuscul Dis. 2023;10(6):xx-xx
