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Pediatric Transplantation|December 22, 2025
Heart Transplantation and Ventricular Assist Device in Duchenne Muscular Dystrophy: A New EraDavid N Rosenthal, Antonio Amodeo, Russell J Butterfield, et al.
Neuromuscular Disorders : NMD|September 26, 2025
Consensus recommendations and considerations for the delivery and monitoring of gene therapy in patients with Duchenne muscular dystrophyJodi M Wolff, Nora Capocci, Evrim Atas, et al.
Contemporary Clinical Trials|April 29, 2017
Developing standardized corticosteroid treatment for Duchenne muscular dystrophyMichela Guglieri, Kate Bushby, Michael P McDermott, et al.
Nature Cancer|February 28, 2022
A human breast cancer-derived xenograft and organoid platform for drug discovery and precision oncologyKatrin P Guillen, Maihi Fujita, Andrew J Butterfield, 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.
Medrxiv : the Preprint Server for Health Sciences|April 8, 2024
The recurrent deep intronic pseudoexon-inducing variant COL6A1 c.930+189C>T results in a consistently severe phenotype of COL6-related dystrophy: Towards clinical trial readiness for splice-modulating therapyA Reghan Foley, Véronique Bolduc, Fady Guirguis, et al.
Brain : a Journal of Neurology|April 3, 2025
Characterization of severe COL6-related dystrophy due to the recurrent variant COL6A1 c.930+189C>TA Reghan Foley, Véronique Bolduc, Fady Guirguis, 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.
Global Change Biology|January 1, 2020
TRY plant trait database - enhanced coverage and open accessJens Kattge, Gerhard Bönisch, Sandra Díaz, et al.
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