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Neuromuscular Disorders : NMD|May 31, 2016
Idebenone reduces respiratory complications in patients with Duchenne muscular dystrophyCraig M McDonald, Thomas Meier, Thomas Voit, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics|June 15, 2019
Maternal copy-number variations in the DMD gene as secondary findings in noninvasive prenatal screeningNathalie Brison, Jazz Storms, Darine Villela, et al.
Pediatric Pulmonology|August 30, 2016
Treatment effect of idebenone on inspiratory function in patients with Duchenne muscular dystrophyGunnar M Buyse, Thomas Voit, Ulrike Schara, et al.
Journal of Neuromuscular Diseases|August 23, 2021
A Combined Prospective and Retrospective Comparison of Long-Term Functional Outcomes Suggests Delayed Loss of Ambulation and Pulmonary Decline with Long-Term Eteplirsen TreatmentOlga Mitelman, Hoda Z Abdel-Hamid, Barry J Byrne, et al.
American Journal of Human Genetics|August 23, 2002
Mutations of the selenoprotein N gene, which is implicated in rigid spine muscular dystrophy, cause the classical phenotype of multiminicore disease: reassessing the nosology of early-onset myopathiesAna Ferreiro, Susana Quijano-Roy, Claire Pichereau, et al.
Neuromuscular Disorders : NMD|September 7, 2020
Gain and loss of abilities in type II SMA: A 12-month natural history studyGiorgia Coratti, Simona Lucibello, Maria C Pera, et al.
Plos One|June 27, 2025
Predicting trajectories of the north star ambulatory assessment total score in Duchenne muscular dystrophyFrancesco Muntoni, James Signorovitch, Nathalie Goemans, et al.
Neuromuscular Disorders : NMD|April 9, 2022
Real-world and natural history data for drug evaluation in Duchenne muscular dystrophy: suitability of the North Star Ambulatory Assessment for comparisons with external controlsFrancesco Muntoni, James Signorovitch, Gautam Sajeev, et al.
Plos One|July 10, 2024
Meaningful changes in motor function in Duchenne muscular dystrophy (DMD): A multi-center studyFrancesco Muntoni, James Signorovitch, Gautam Sajeev, et al.
European Journal of Medical Genetics|October 11, 2025
Re-evaluating acceptable risk of death from gene therapy: A threshold study among individuals with Duchenne muscular dystrophy and their caregivers in the US and UKHolly Peay, Ryan Fischer, Megan McNiff, et al.
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