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Neurology. Clinical Practice|June 27, 2024
Therapeutic Role of Nusinersen on Respiratory Progression in Pediatric Patients With Spinal Muscular Atrophy Type 2 and Nonambulant Type 3Federica Trucco, Deborah Ridout, Harriet Weststrate, et al.Annals of Neurology|September 14, 2020
Clinical Variability in Spinal Muscular Atrophy Type IIIGiorgia Coratti, Sonia Messina, Simona Lucibello, et al.Journal of Neurology, Neurosurgery, and Psychiatry|January 30, 2026
Loss of ambulation in SMA III at the time of disease-modifying treatments: an international studyGiorgia Coratti, Francesca Bovis, Valentina Franchino, et al.Microbiology Resource Announcements|May 16, 2020
Complete Genome Sequences of Cluster G Mycobacteriophage Darionha, Cluster A Mycobacteriophage Salz, and Cluster J Mycobacteriophage ThreeRngTarjayAndrea M Sandoval, Amber M Abram, Zahraa M Alhabib, et al.Contemporary Clinical Trials|April 29, 2017
Developing standardized corticosteroid treatment for Duchenne muscular dystrophyMichela Guglieri, Kate Bushby, Michael P McDermott, et al.Trials|May 26, 2018
A checklist for clinical trials in rare disease: obstacles and anticipatory actions-lessons learned from the FOR-DMD trialRebecca A Crow, Kimberly A Hart, Michael P McDermott, et al.Neurology|February 9, 2024
Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy: A Randomized Controlled TrialUtkarsh J Dang, Jesse M Damsker, Michela Guglieri, et al.JAMA Neurology|August 29, 2022
Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical TrialMichela Guglieri, Paula R Clemens, Seth J Perlman, et al.Human Mutation|December 3, 2021
The clinical and molecular spectrum of QRICH1 associated neurodevelopmental disorderSmitha Kumble, Amanda M Levy, Jaya Punetha, et al.JAMA|April 5, 2022
Effect of Different Corticosteroid Dosing Regimens on Clinical Outcomes in Boys With Duchenne Muscular Dystrophy: A Randomized Clinical TrialMichela Guglieri, Kate Bushby, Michael P McDermott, et al.Pageof 38