Autori: Pedemonte M

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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

Early treatment of type II SMA slows rate of progression of scoliosis

Type I spinal muscular atrophy patients treated with nusinersen: 4-year follow-up of motor, respiratory and bulbar function

Long term follow-up of scoliosis progression in type II SMA patients

Nusinersen efficacy data for 24-month in type 2 and 3 spinal muscular atrophy

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

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

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

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