Autori: Baranello G

Filtra

Data

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

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

Risdiplam in Type 1 Spinal Muscular Atrophy

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

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

Gain and loss of upper limb abilities in Duchenne muscular dystrophy patients: A 24-month study

Development of an International SMA Bulbar Assessment for Inter-professional Administration

Longitudinal Analysis of PUL 2.0 Domains in Ambulant and Non-Ambulant Duchenne Muscular Dystrophy Patients: How do they Change in Relation to Functional Ability?

2-Year Change in Revised Hammersmith Scale Scores in a Large Cohort of Untreated Paediatric Type 2 and 3 SMA Participants

Revised upper limb module in type II and III spinal muscular atrophy: 24-month changes

Torna in alto