Allison Grange

Efficacy and safety of risdiplam in patients with type 1 spinal muscular atrophy: a 3-year open-label extension of the two-part, phase 2 FIREFISH trial

Maria Mazurkiewicz-Bełdzińska, Maria Mazurkiewicz-Bełdzińska, Laurent Servais, Giovanni Baranello et autres

pl, gb, fr, us, be, ch, it, au, ru, cn, br (code pays fourni par la source)

Identification of Novel Smartphone-Based Digital Biomarkers to Characterize Lower and Upper Limb Motor Functions in Patients with Duchenne Muscular Dystrophy (P3-11.005)

Shotaro Tachibana, Allison Grange, Dominique Vincent-Genod, Adeline Allara et autres

To assess the feasibility of smartphone-based digital biomarkers (dBMKs) to objectively measure lower and upper limb motor functions in patients with Duchenne muscular dystrophy (DMD).

fr, ca (code pays fourni par la source)

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, Laurent Servais et autres

it, pl, au, be, fr, gb, cn, br, us, ch, ru (code pays fourni par la source)

Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial

Eugenio Mercuri, Nicolas Deconinck, Elena Mazzone, A. Nascimento et autres

it, us, be, es, ca, jp, fr, gb, de, pl, ch (code pays fourni par la source)