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Lancet Neurol. 2018 Mar;17(3):251-267. doi: 10.1016/S1474-4422(18)30024-3. Epub 2018 Feb 3.

Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management.

Author information

1
Department of Pediatrics, MetroHealth Medical Center, Case Western Reserve University, Cleveland, OH, USA. Electronic address: dbirnkrant@metrohealth.org.
2
John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Newcastle University, Newcastle upon Tyne, UK.
3
RTI International, Research Triangle Park, NC, USA.
4
Department of Rehabilitation Medicine, Seattle Children's Hospital, Seattle, WA, USA.
5
Section of Pediatric Gastroenterology, Hepatology, and Nutrition, Children's Hospital Colorado, Aurora, CO, USA.
6
Doctor of Physical Therapy Division, Department of Orthopaedics, Duke University School of Medicine, Durham, NC, USA.
7
Department of Neurology, University of Pittsburgh School of Medicine, and Neurology Service, Department of Veterans Affairs Medical Center, Pittsburgh, PA, USA.
8
Division of Endocrinology and Metabolism, Children's Hospital of Eastern Ontario, and University of Ottawa, Ottawa, ON, Canada.
9
School of Medicine and Dentistry, University of Rochester, Rochester, NY, USA.
10
Rare Disorders and Health Outcomes Team, National Center on Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, Atlanta, GA, USA.
11
Medical Nutrition Consulting of Media LLC, and Children's Hospital of Philadelphia, Philadelphia, PA, USA.
12
Center for Genetic Muscle Disorders, Kennedy Krieger Institute, and Johns Hopkins School of Medicine, Baltimore, MD, USA.
13
Division of Endocrinology and Diabetes, Golisano Children's Hospital, University of Rochester Medical Center, Rochester, NY, USA.

Erratum in

Abstract

Since the publication of the Duchenne muscular dystrophy (DMD) care considerations in 2010, multidisciplinary care of this severe, progressive neuromuscular disease has evolved. In conjunction with improved patient survival, a shift to more anticipatory diagnostic and therapeutic strategies has occurred, with a renewed focus on patient quality of life. In 2014, a steering committee of experts from a wide range of disciplines was established to update the 2010 DMD care considerations, with the goal of improving patient care. The new care considerations aim to address the needs of patients with prolonged survival, to provide guidance on advances in assessments and interventions, and to consider the implications of emerging genetic and molecular therapies for DMD. The committee identified 11 topics to be included in the update, eight of which were addressed in the original care considerations. The three new topics are primary care and emergency management, endocrine management, and transitions of care across the lifespan. In part 1 of this three-part update, we present care considerations for diagnosis of DMD and neuromuscular, rehabilitation, endocrine (growth, puberty, and adrenal insufficiency), and gastrointestinal (including nutrition and dysphagia) management.

PMID:
29395989
PMCID:
PMC5869704
DOI:
10.1016/S1474-4422(18)30024-3
[Indexed for MEDLINE]
Free PMC Article

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