Riccardo Masson

Risdiplam in Type 1 Spinal Muscular Atrophy

Giovanni Baranello, Basil T. Darras, John Day et al. · New England Journal of Medicine · 2021 · 383 citations · Full text

Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls

Basil T. Darras, Riccardo Masson, Maria Mazurkiewicz-Bełdzińska et al. · New England Journal of Medicine · 2021 · 262 citations · Full text

Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial

Eugenio Mercuri, Francesco Muntoni, Giovanni Baranello et al. · The Lancet Neurology · 2021 · 237 citations

Gene Therapies, Clinical Trials, Neurology +2

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

Eugenio Mercuri, Juan J. Vílchez, Odile Boespflug‐Tanguy et al. · The Lancet Neurology · 2024 · 100 citations

Duchenne Muscular Dystrophy, Clinical Trials, Pharmacotherapy +2

Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial

Riccardo Masson, Maria Mazurkiewicz-Bełdzińska, Kristy Rose et al. · The Lancet Neurology · 2022 · 96 citations · Full text

Type 1, Firefish Part 2, Rehabilitation +6