C. Sobreira

A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in Duchenne muscular dystrophy

J. J. Padilla, C. Palermo, M. Pane, 2018, Neuromuscular Disorders.

Expanding the phenotypes of the Pro56Ser VAPB mutation: Proximal SMA with dysautonomia

M. Zago, W. Marques, P. Abou-Sleiman, 2006, Muscle & nerve.

Leigh Syndrome Due to mtDNA Pathogenic Variants

L. Vilarinho, L. Vedolin, F. Vairo, 2019, Journal of Inborn Errors of Metabolism and Screening.

Mitochondrial encephalomyopathy with coenzyme Q10 deficiency

S. Dimauro, D. Mojon, F. Santorelli, 1997, Neurology.

A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in Duchenne muscular dystrophy

C. Palermo, M. Pane, E. Mercuri, 2018, Neuromuscular Disorders.