Elena S. Mazzone
Rome, Italy
Last Updated: 09/26/2026
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Found 65 publications
A plain language summary of the SAPPHIRE clinical trial of apitegromab in children and young adults with spinal muscular atrophy.
Journal: Expert review of neurotherapeutics
Published: November 13, 2025
Refining functional phenotypes in an international cohort of untreated paediatric type 2 and 3 SMA patients using the Revised Hammersmith Scale.
Journal: Neuromuscular disorders : NMD
Published: October 10, 2025
Safety and efficacy of apitegromab in nonambulatory type 2 or type 3 spinal muscular atrophy (SAPPHIRE): a phase 3, double-blind, randomised, placebo-controlled trial.
Journal: The Lancet. Neurology
Published: March 25, 2025
Long-term efficacy, safety, and patient-reported outcomes of apitegromab in patients with spinal muscular atrophy: results from the 36-month TOPAZ study.
Journal: Frontiers in neurology
Published: April 18, 2024
Disease Trajectories in the Revised Hammersmith Scale in a Cohort of Untreated Patients with Spinal Muscular Atrophy types 2 and 3.
Journal: Journal of neuromuscular diseases
Published: March 01, 2024
Gain and loss of upper limb abilities in Duchenne muscular dystrophy patients: A 24-month study.
Journal: Neuromuscular disorders : NMD
Published: August 18, 2023
Patient reported outcome measure for upper limb in Duchenne muscular dystrophy: correlation with PUL2.0.
Journal: Neuromuscular disorders : NMD
Published: April 18, 2023
Correction to: Two‑year efficacy and safety of risdiplam in patients with type 2 or non‑ambulant type 3 spinal muscular atrophy (SMA).
Journal: Journal of neurology
Published: April 18, 2023
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?
Journal: Journal of neuromuscular diseases
Published: April 17, 2023
2-Year Change in Revised Hammersmith Scale Scores in a Large Cohort of Untreated Paediatric Type 2 and 3 SMA Participants.
Journal: Journal of clinical medicine
Published: January 20, 2023
Emergencies cards for neuromuscular disorders 1st Consensus Meeting from UILDM - Italian Muscular Dystrophy Association Workshop report.
Journal: Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology
Published: October 26, 2022
Last Updated: 09/26/2026