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Plos One|May 3, 2019
Gene therapy as a potential therapeutic option for Duchenne muscular dystrophy: A qualitative preference study of patients and parentsHolly Landrum Peay, Ryan Fischer, Janice P Tzeng, et al.Journal of Neurology|January 12, 2017
Reduced serum myostatin concentrations associated with genetic muscle disease progressionPeter M Burch, Oksana Pogoryelova, Joe Palandra, et al.BMC Neurology|May 21, 2020
Longitudinal functional and imaging outcome measures in FKRP limb-girdle muscular dystrophyDoris G Leung, Alex E Bocchieri, Shivani Ahlawat, et al.Mabs|September 15, 2016
Beyond CDR-grafting: Structure-guided humanization of framework and CDR regions of an anti-myostatin antibodyJames R Apgar, Michelle Mader, Rita Agostinelli, et al.Muscle & Nerve|May 7, 2021
A phase Ib/IIa, open-label, multiple ascending-dose trial of domagrozumab in fukutin-related protein limb-girdle muscular dystrophyDoris G Leung, Alex E Bocchieri, Shivani Ahlawat, et al.Molecular Genetics & Genomic Medicine|March 23, 2021
Patients' and caregivers' maximum acceptable risk of death for non-curative gene therapy to treat Duchenne muscular dystrophyHolly L Peay, Ryan Fischer, Brennan Mange, et al.Human Gene Therapy|January 25, 2023
Microdystrophin Expression as a Surrogate Endpoint for Duchenne Muscular Dystrophy Clinical TrialsJeffrey S Chamberlain, Melissa Robb, Serge Braun, et al.Orphanet Journal of Rare Diseases|May 11, 2019
Priorities when deciding on participation in early-phase gene therapy trials for Duchenne muscular dystrophy: a best-worst scaling experiment in caregivers and adult patientsRyan S Paquin, Ryan Fischer, Carol Mansfield, et al.Human Molecular Genetics|January 3, 2018
Myostatin inhibition using mRK35 produces skeletal muscle growth and tubular aggregate formation in wild type and TgACTA1D286G nemaline myopathy miceJennifer A Tinklenberg, Emily M Siebers, Margaret J Beatka, et al.Journal of Neuromuscular Diseases|April 24, 2026
TREAT-NMD advisory committee for therapeutics: Preclinical and clinical learnings from 15 years of TACTAnnemieke Aartsma-Rus, Laura Robertson, Lindsay N Alfano, et al.Pageof 4