COST Actions: fostering collaborative research for rare diseases
Desviat LR, Mallebrera CJ, Vallejo-Illarramendi A, Mayan MD, Nogales-Gadea G, Arechavala-Gomeza V. COST Actions: fostering collaborative research for rare diseases.…
Optimization of antisense-mediated exon skipping for Duchenne muscular dystrophy
Dzierlega K, Yokota T. Optimization of antisense-mediated exon skipping for Duchenne muscular dystrophy. Gene Ther 2020;27:407-416 Abstract Duchenne muscular…
Urinary incontinence in men with Duchenne and Becker muscular dystrophy
Morse CI, Higham K, Bostock EL, Jacques MF. Urinary incontinence in men with Duchenne and Becker muscular dystrophy. Plos One…
Cognitive Deficits in Myopathies
Peristeri E, Aloizou AM, Keramida P, et al. Cognitive Deficits in Myopathies. International journal of molecular sciences 2020;21 Abstract…
Safety issues and harmful pharmacological interactions of nutritional supplements in Duchenne muscular dystrophy: considerations for Standard of Care and emerging virus outbreaks
Boccanegra B, Verhaart IEC, Cappellari O, Vroom E, De Luca A. Safety issues and harmful pharmacological interactions of nutritional supplements…
Overview of viltolarsen (NS-065/NCNP-01) clinical trials
Trial publications Phase I open-label trial (NCT02081625) Exploratory Study of NS-065/NCNP-01 in DMD Komaki H, Nagata T, Saito T, et…
Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A Phase 2 Randomized Clinical Trial
Clemens PR, Rao VK, Connolly AM, et al. Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy…
EMQN best practice guidelines for genetic testing in dystrophinopathies
Fratter C, Dalgleish R, Allen SK, Santos R, Abbs S, Tuffery-Giraud S, et al. EMQN best practice guidelines for genetic…
Exposure-Response Analysis of Vamorolone (VBP15) in Boys With Duchenne Muscular Dystrophy
Li X, Conklin LS, van den Anker J, Hoffman EP, Clemens PR, Jusko WJ. Exposure-Response Analysis of Vamorolone (VBP15) in…
Intraoperative neurophysiological monitoring during scoliosis surgery in patients with Duchenne muscular dystrophy
Dulfer SE, Lange F, Wapstra FH, et al. Intraoperative neurophysiological monitoring during scoliosis surgery in patients with Duchenne muscular dystrophy.…
