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Neuromuscular Disorders : NMD|November 10, 2019
Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE studyDarryl C De Vivo, Enrico Bertini, Kathryn J Swoboda, et al.
Neurology|October 17, 2020
Respiratory Trajectories in Type 2 and 3 Spinal Muscular Atrophy in the iSMAC Cohort StudyFederica Trucco, Deborah Ridout, Mariacristina Scoto, et al.
Annals of Clinical and Translational Neurology|June 24, 2021
Nusinersen in pediatric and adult patients with type III spinal muscular atrophyMaria Carmela Pera, Giorgia Coratti, Francesca Bovis, et al.
Journal of Cerebral Blood Flow and Metabolism : Official Journal of the International Society of Cerebral Blood Flow and Metabolism|August 30, 2008
The role of animal models in evaluating reasonable safety and efficacy for human trials of cell-based interventions for neurologic conditionsAlan Regenberg, Debra J H Mathews, David M Blass, et al.
JAMA Network Open|January 25, 2022
Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A 30-Month Nonrandomized Controlled Open-Label Extension TrialJean K Mah, Paula R Clemens, Michela Guglieri, et al.
Annals of Neurology|May 14, 2020
GGPS1 Mutations Cause Muscular Dystrophy/Hearing Loss/Ovarian Insufficiency SyndromeA Reghan Foley, Yaqun Zou, James E Dunford, et al.
Journal of Clinical Medicine|March 11, 2023
2-Year Change in Revised Hammersmith Scale Scores in a Large Cohort of Untreated Paediatric Type 2 and 3 SMA ParticipantsGeorgia Stimpson, Danielle Ramsey, Amy Wolfe, et al.
Plos One|April 5, 2024
Strategies for primary HPV test-based cervical cancer screening programme in resource-limited settings in India: Results from a quasi-experimental pragmatic implementation trialAnu Mary Oommen, Rita Isaac, Biswajit Paul, et al.
European Journal of Neurology|June 27, 2025
Longitudinal Assessment of 4-Year HFMSE Changes in SMA II and III Patients Treated With NusinersenGiorgia Coratti, Francesca Bovis, Marika Pane, et al.
Neurology|August 28, 2019
Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle functionEric P Hoffman, Benjamin D Schwartz, Laurel J Mengle-Gaw, et al.
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