J. C. Oferil

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.

GDF-15 Is Elevated in Children with Mitochondrial Diseases and Is Induced by Mitochondrial Dysfunction

F. Villarroya, S. Kalko, E. Ruiz-Pesini, 2016, PloS one.

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.