Raggruppamento Primario: Malattie Neuromuscolari

Filtra

Data

The relationship between deficit in digit span and genotype in nonsense mutation Duchenne muscular dystrophy

Ataluren treatment of patients with nonsense mutation dystrophinopathy.

Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial

Management of motor rehabilitation in individuals with muscular dystrophies. 1(st) Consensus Conference report from UILDM – Italian Muscular Dystrophy Association (Rome, January 25-26, 2019)

Natural history of Type 1 spinal muscular atrophy: a retrospective, global, multicenter study

Patient and parent oriented tools to assess health-related quality of life, activity of daily living and caregiver burden in SMA. Rome, 13 July 2019

First international workshop on rehabilitation management and clinical outcome measures for spinal muscular atrophy

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

A 5-center experience with intrathecal administration of nusinersen in SMA1 in Italy letter to the editor of european journal of pediatric neurology regarding the manuscript “single-center experience with intrathecal administration of nusinersen in children with spinal muscular atrophy type 1″ written by pechmann and colleagues”

Expanded access program with Nusinersen in SMA type I in Italy: Strengths and pitfalls of a successful experience

Torna in alto