Autori: Bruno C

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

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

Correction to: Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study

Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study

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

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

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

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

Upper Limb Changes in DMD Patients Amenable to Skipping Exons 44, 45, 51 and 53: A 24-Month Study

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