Tracey Willis

Safety and efficacy of tamoxifen in boys with Duchenne muscular dystrophy (TAMDMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

M. Main, A. Manzur, A. Childs, 2023, The Lancet Neurology.

Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

G. Acsadi, M. Scoto, E. Finanger, 2024, The Lancet Neurology.

Therapeutic Role of Nusinersen on Respiratory Progression in Pediatric Patients With Spinal Muscular Atrophy Type 2 and Nonambulant Type 3.

M. Scoto, Claudio Bruno, D. D. De Vivo, 2024, Neurology. Clinical practice.

Risdiplam in Spinal Muscular Atrophy: Safety Profile and Use Through The Early Access to Medicine Scheme for the Paediatric Cohort in Great Britain

M. Scoto, E. Wraige, D. Parasuraman, 2024, Journal of neuromuscular diseases.