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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

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

Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls

Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial

Communicative development inventory in type 1 and presymptomatic infants with spinal muscular atrophy: a cohort study

Erratum: Transcranial optical monitoring for detecting intracranial pressure alterations in children with benign external hydrocephalus: a proof-of-concept study

Neurocognitive profile of a cohort of SMA type 1 pediatric patients and emotional aspects, resilience and coping strategies of their caregivers

Psychometric Properties and Validation of the Italian Version of Ages & Stages Questionnaires Third Edition

Translation and Cultural Adaptation of the StimQ for Use with Italian Children from Kindergartens

Oral and Swallowing Abilities Tool (OrSAT) in nusinersen treated patients

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