Autori: Bertini E

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The nonsense mutation stop+4 model correlates with motor changes in Duchenne muscular dystrophy

The Spinal Muscular Atrophy Health Index: Italian validation of a disease-specific outcome measure

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

Diagnostic journey in Spinal Muscular Atrophy: Is it still an odyssey?

Respiratory Needs in Patients with Type 1 Spinal Muscular Atrophy Treated with Nusinersen

Longitudinal natural history in young boys with Duchenne muscular dystrophy

Nusinersen in type 1 spinal muscular atrophy: Twelve-month real-world data

Long-term natural history data in Duchenne muscular dystrophy ambulant patients with mutations amenable to skip exons 44, 45, 51 and 53

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