Library

Buyse GM, Goemans N, van den Hauwe M, et al. Idebenone as a novel, therapeutic approach for Duchenne muscular dystrophy:…

Servais L, Straathof CSM, Schara U, et al. Long-term data with idebenone on respiratory function outcomes in patients with Duchenne…

Siegel BI, Cakmak A, Reinertsen E, et al. Use of a wearable device to assess sleep and motor function in…

Motoki T, Shimizu-Motohashi Y, Saito I, et al. Renal dysfunction can occur in advanced-stage Duchenne muscular dystrophy. Muscle & nerve…

Ling C, Dai Y, Fang L, et al. Exonic rearrangements in DMD in Chinese Han individuals affected with Duchenne and…

Lao YE, Molden E, Kringen MK, et al. Fatal liver failure after therapeutic doses of paracetamol in a patient with…

Asher DR, Thapa K, Dharia SD, et al. Clinical development on the frontier: gene therapy for duchenne muscular dystrophy. Expert…

McPherson AC, Amin R, McAdam L, Kalnins D, Lui T. Growth assessment and weight management in paediatric neuromuscular clinics: a…

Li D, McDonald CM, Elfring GL, et al. Assessment of Treatment Effect With Multiple Outcomes in 2 Clinical Trials of…

Aartsma-Rus A, Corey DR. The 10th Oligonucleotide Therapy Approved: Golodirsen for Duchenne Muscular Dystrophy. Nucleic acid therapeutics 2020;30:67-70   Introduction…