Journal: Neuromuscular disorders: NMD

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Safety, tolerability and pharmacokinetics of eteplirsen in young boys aged 6-48 months with Duchenne muscular dystrophy amenable to exon 51 skipping

Bulbar function in spinal muscular atrophy (SMA): State of art and new challenges. 21st July 2023, Rome, Italy

Changes in abilities over the initial 12 months of nusinersen treatment for type II SMA.

Upper limb function changes over 12 months in untreated SMA II and III individuals: an item-level analysis using the Revised Upper Limb Module

Italian survey on evolving SMA care with disease-modifying therapies: a consensus workshop on nutrition, swallowing, respiratory and rehabilitation care

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

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

Evolution of neuropsychological and behavioral profile in a cohort of pediatric patients with Becker muscular dystrophy in a longitudinal study

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

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