Autori: Scoto M

Filtra

Data

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

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

Different trajectories in upper limb and gross motor function in spinal muscular atrophy

Nusinersen in pediatric and adult patients with type III spinal muscular atrophy

Respiratory Trajectories in Type 2 and 3 Spinal Muscular Atrophy in the iSMAC Cohort Study

Clinical Variability in Spinal Muscular Atrophy Type III

Age and baseline values predict 12 and 24-month functional changes in type 2 SMA

Development of an academic disease registry for spinal muscular atrophy

Revised upper limb module for spinal muscular atrophy: 12?month changes

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