Autori: Pini A

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Data

Reliability of the Hammersmith functional motor scale for spinal muscular atrophy in a multicentric study

The Hammersmith functional score correlates with the SMN2 copy number: a multicentric study

Randomized, double-blind, placebo-controlled trial of phenylbutyrate in spinal muscular atrophy

Association between Reported Sleep Disorders and Behavioral Issues in Children with Myotonic Dystrophy Type 1-Results from a Retrospective Analysis in Italy

Parental diagnostic delay and developmental outcomes in congenital and childhood-onset myotonic dystrophy type 1

Natural history of Becker muscular dystrophy: DMD gene mutations predict clinical severity

Prevalence of Duchenne muscular dystrophy in Italy: a nationwide survey

Type I spinal muscular atrophy and disease modifying treatments: a nationwide study in children born since 2016

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

Onasemnogene abeparvovec in spinal muscular atrophy: predictors of efficacy and safety in na‹ve patients with spinal muscular atrophy and following switch from other therapies

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