Autori: Vita G

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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

Age, corticosteroid treatment and site of mutations affect motor functional changes in young boys with Duchenne Muscular Dystrophy

North Star Ambulatory Assessment changes in ambulant Duchenne boys amenable to skip exons 44, 45, 51, and 53: A 3 year follow up

Type I SMA “new natural history”: long-term data in nusinersen-treated patients

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

Psychosocial impact of sport activity in neuromuscular disorders.

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

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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