Journal: Neuromuscular Disorders

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

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

Functional outcome measures in young, steroid-na‹ve boys with Duchenne muscular dystrophy

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

Age related treatment effect in type II Spinal Muscular Atrophy pediatric patients treated with nusinersen

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

A critical review of patient and parent caregiver oriented tools to assess health-related quality of life, activity of daily living and caregiver burden in spinal muscular atrophy

Intrathecal administration of nusinersen in adult and adolescent patients with spinal muscular atrophy and scoliosis: Transforaminal versus conventional approach

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