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Nature Medicine|June 17, 2022
Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trialKevin A Strauss, Michelle A Farrar, Francesco Muntoni, et al.
Nature Medicine|June 17, 2022
Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trialKevin A Strauss, Michelle A Farrar, Francesco Muntoni, et al.
Neurology|March 19, 2020
Modeling disease trajectory in Duchenne muscular dystrophyWilliam D Rooney, Yosef A Berlow, William T Triplett, et al.
Annals of Clinical and Translational Neurology|May 15, 2026
Early Clinical, Imaging, and Pathological Characteristics of SRPK3/TTN-Digenic MyopathyRotem Orbach, Sandra Donkervoort, Carola Hedberg-Oldfors, et al.
Plos One|February 22, 2017
Revised Hammersmith Scale for spinal muscular atrophy: A SMA specific clinical outcome assessment toolDanielle Ramsey, Mariacristina Scoto, Anna Mayhew, et al.
Pediatrics|January 18, 2012
Pediatric analgesic clinical trial designs, measures, and extrapolation: report of an FDA scientific workshopCharles B Berde, Gary A Walco, Elliot J Krane, et al.
Muscle & Nerve|June 27, 2015
Old measures and new scores in spinal muscular atrophy patientsElena Mazzone, Jacqueline Montes, Marion Main, et al.
The Journals of Gerontology. Series A, Biological Sciences and Medical Sciences|March 3, 2019
A Multi-study Coordinated Meta-analysis of Pulmonary Function and Cognition in AgingEmily C Duggan, Andrea M Piccinin, Sean Clouston, et al.
Psychology of Addictive Behaviors : Journal of the Society of Psychologists in Addictive Behaviors|January 9, 2025
Genetic and environmental influences on alcohol consumption in middle to late lifeDaniel E Gustavson, Tyler R Bell, Erik J Buchholz, et al.
Biorxiv : the Preprint Server for Biology|October 7, 2020
SARS-CoV-2 infected cells present HLA-I peptides from canonical and out-of-frame ORFsShira Weingarten-Gabbay, Susan Klaeger, Siranush Sarkizova, et al.
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