Yulia Papina

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)

Rare SMA Patients: A Comprehensive Look at Clinical Features, Genetic Profiles and Therapeutic Approaches

Kristina Mikhalchuk, Svetlana Artemieva, Viktoria Zabnenkova, Maria Akhkiamova et autres

Spinal muscular atrophy 5q (5q SMA) is one of the most prevalent autosomal recessive disorders globally. The underlying cause of 5q SMA is attributed to variants in SMN1. To date, there are no reported cases of gene-based therapy in rare patients with …

ru (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 J. 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 Maria Mercuri, Nicolas Deconinck, Elena Stacy Mazzone, Andrés Nascimento et autres

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