Autori: Tiziano FD

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Data

Clinical Phenotype of Pediatric and Adult Patients With Spinal Muscular Atrophy With Four SMN2 Copies: Are They Really All Stable?

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

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

SMA-miRs (miR-181a-5p, -324-5p, and -451a) are overexpressed in spinal muscular atrophy skeletal muscle and serum samples

Type I SMA “new natural history”: long-term data in nusinersen-treated patients

Nusinersen in type 1 spinal muscular atrophy: Twelve-month real-world data

Nusinersen in type 1 SMA infants, children and young adults: Preliminary results on motor function

Clinical phenotypes and trajectories of disease progression in type 1 spinal muscular atrophy

Pilot trial of phenylbutyrate in spinal muscular atrophy

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