Raggruppamento Primario: Malattie Neuromuscolari

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Data

Switching therapies: safety profile of Onasemnogene abeparvovec-xioi in a SMA1 patient previously treated with Risdiplam.

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

Language Development in Preschool Duchenne Muscular Dystrophy Boys

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

Identification of a cytokine profile in serum and cerebrospinal fluid of pediatric and adult spinal muscular atrophy patients and its modulation upon nusinersen treatment

Neurological assessment of newborns with spinal muscular atrophy identified through neonatal screening

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

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

Predictive models in SMA II natural history trajectories using machine learning: A proof of concept study

Body mass index in type 2 spinal muscular atrophy: a longitudinal study

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