Functional outcome measures in young, steroid-naïve boys with Duchenne muscular dystrophy
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Published:2022-06
Issue:6
Volume:32
Page:460-467
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ISSN:0960-8966
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Container-title:Neuromuscular Disorders
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language:en
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Short-container-title:Neuromuscular Disorders
Author:
Mayhew Anna GORCID, Moat Dionne, McDermott Michael P., Eagle Michelle, Griggs Robert C., King Wendy M., James Meredith K.ORCID, Muni-Lofra Robert, Shillington Alison, Gregson SarahORCID, Pallant Lindsey, Skura ChristyORCID, Staudt Loretta A.ORCID, Eichinger Katy, McMurchie Heather, Rabb Rosanna, Di Marco Marina, Brown Sarah, Zanin Riccardo, Arnoldi Maria Teresa, McIntyre Melissa, Wilson AmeliaORCID, Alfano Lindsay N.ORCID, Lowes Linda P.ORCID, Blomgren Colleen, Milev Evelin, Iodice Mario, Pasternak Amy, Chiu Angela, Lehnert Ilka, Claus Nicole, Dieruf Kathy A.ORCID, Rolle Enrica, Nicorici Alina, Andres Barbara, Hobbiebrunken Elke, Roetmann Gerda, Kern Victoria, Civitello Matthew, Vogt Sibylle, Hayes Melissa J.ORCID, Scholtes CherylORCID, Lacroix CatherineORCID, Gunn Tara, Warner Sinead, Newman JenniferORCID, Barp Andrea, Kundrat Katherine, Kovelman StaciORCID, Powers Penny J., Guglieri Michela
Funder
Parent Project Muscular Dystrophy PTC Therapeutics Inc Sarepta Therapeutics Muscular Dystrophy Association Fondazione Telethon Foundation for the National Institutes of Health
Subject
Genetics (clinical),Neurology (clinical),Neurology,Pediatrics, Perinatology and Child Health
Reference30 articles.
1. Evidence-based path to newborn screening for Duchenne muscular dystrophy;Mendell;Ann Neurol,2012 2. Diagnosis and management of Duchenne muscular dystrophy, part 3: primary care, emergency management, psychosocial care, and transitions of care across the lifespan;Birnkrant;Lancet Neurol,2018 3. Improving recognition of Duchenne muscular dystrophy: a retrospective case note review;van Ruiten;Arch Dis Child,2014 4. Delayed diagnosis in Duchenne muscular dystrophy: data from the muscular dystrophy surveillance, tracking, and research network (MD STARnet);Ciafaloni;J Pediatr,2009 5. Is there a delay in diagnosis of Duchenne muscular dystrophy among preterm-born males?;Soim;J Child Neurol,2018
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