Y. Takeshima

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.

Nusinersen versus Sham Control in Infantile‐Onset Spinal Muscular Atrophy

R. J. Ramamurthi, S. Y. Kim, H. Y. Wang, 2017, The New England journal of medicine.

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.