Overview of eteplirsen (AVI-4658/Exondys51) clinical trials
Trial publications Phase I local treatment (NCT00159250) Safety and Efficacy Study of Antisense Oligonucleotides in Duchenne Muscular Dystrophy • Kinali M,…
The use of intravenous bisphosphonate therapy to treat vertebral fractures due to osteoporosis among boys with Duchenne muscular dystrophy
Sbrocchi AM, Rauch F, Jacob P, et al. The use of intravenous bisphosphonate therapy to treat vertebral fractures due to…
Heart rate reduction strategy using ivabradine in end-stage Duchenne cardiomyopathy
Adorisio R, Calvieri C, Cantarutti N, et al. Heart rate reduction strategy using ivabradine in end-stage Duchenne cardiomyopathy. Int J…
Nonclinical Exon Skipping Studies with 2'-O-Methyl Phosphorothioate Antisense Oligonucleotides in mdx and mdx-utrn-/- Mice Inspired by Clinical Trial Results
van Putten M, Tanganyika-de Winter C, Bosgra S, Aartsma-Rus A. Nonclinical Exon Skipping Studies with 2'-O-Methyl Phosphorothioate Antisense Oligonucleotides in…
Cardiac and skeletal muscle effects in the randomized HOPE-Duchenne trial
Taylor M, Jefferies J, Byrne B, et al. Cardiac and skeletal muscle effects in the randomized HOPE-Duchenne trial. Neurology 2019…
Speckle-Tracking Echocardiography in Children With Duchenne Muscular Dystrophy: A Prospective Multicenter Controlled Cross-Sectional Study
Amedro P, Vincenti M, De La Villeon G, et al. Speckle-Tracking Echocardiography in Children With Duchenne Muscular Dystrophy: A Prospective…
Evaluation of Left-Sided Heart Chambers With Novel Echocardiographic Techniques in Men With Duchenne or Becker Muscular Dystrophy
Cirino RHD, Scola RH, Ducci RD, et al. Evaluation of Left-Sided Heart Chambers With Novel Echocardiographic Techniques in Men With…
Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy
Amoasii L, Hildyard JCW, Li H, et al. Gene editing restores dystrophin expression in a canine model of Duchenne muscular…
Utrophin upregulation by ezutromid (SMT C1100) [terminated]
Ricotti V, Spinty S, Roper H, et al. Safety, Tolerability, and Pharmacokinetics of SMT C1100, a 2-Arylbenzoxazole Utrophin Modulator, following…
Prevalence and long-term monitoring of humoral immunity against adeno-associated virus in Duchenne Muscular Dystrophy patients
Leborgne C, Latournerie V, Boutin S, et al. Prevalence and long-term monitoring of humoral immunity against adeno-associated virus in Duchenne…
