Autori: Bertini E

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Expanded access program with Nusinersen in SMA type I in Italy: Strengths and pitfalls of a successful experience

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”

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

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

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

Longitudinal Analysis of PUL 2.0 Domains in Ambulant and Non-Ambulant Duchenne Muscular Dystrophy Patients: How do they Change in Relation to Functional Ability?

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

2-Year Change in Revised Hammersmith Scale Scores in a Large Cohort of Untreated Paediatric Type 2 and 3 SMA Participants

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

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