Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac, bone health, and orthopaedic management | DuchenneXchange

welcome to DuchenneXchange

- a positively charged Duchenne muscular dystrophy community.
  • join today!
Guides & Guidelines

Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac, bone health, and orthopaedic management

key information

source: The Lancet Neurology

year: 2018

authors: Birnkrant DJ, Bushby K, Bann CM, Apkon SD, Blackwell A, Brumbaugh D, Case LE, Clemens PR, Hadjiyannakis S, Pandya S, Street N, Tomezsko J, Wagner KR, Ward LM, Weber DR, DMD Care Considerations Working Group

summary/abstract:

A coordinated, multidisciplinary approach to care is essential for optimum management of the primary manifestations and secondary complications of Duchenne muscular dystrophy (DMD). Contemporary care has been shaped by the availability of more sensitive diagnostic techniques and the earlier use of therapeutic interventions, which have the potential to improve patients’ duration and quality of life. In part 2 of this update of the DMD care considerations, we present the latest recommendations for respiratory, cardiac, bone health and osteoporosis, and orthopaedic and surgical management for boys and men with DMD. Additionally, we provide guidance on cardiac management for female carriers of a disease-causing mutation. The new care considerations acknowledge the effects of long-term glucocorticoid use on the natural history of DMD, and the need for care guidance across the lifespan as patients live longer. The management of DMD looks set to change substantially as new genetic and molecular therapies become available.

organization: Case Western Reserve University, Cleveland, USA; Newcastle University, Newcastle upon Tyne, UK; RTI International, Research Triangle Park, NC, USA; Seattle Children's Hospital, Seattle, WA, USA; Duke University School of Medicine, Durham, USA; University of Pittsburgh School of Medicine, and Neurology Service, Pittsburgh, USA; Children's Hospital of Eastern Ontario, and University of Ottawa, Canada; University of Rochester, Rochester, NY, USA; Centers for Disease Control and Prevention, Atlanta, GA, USA; Children's Hospital of Philadelphia, Philadelphia, USA; Kennedy Krieger Institute, and Johns Hopkins School of Medicine, Baltimore, USA; Golisano Children's Hospital, University of Rochester Medical Center, Rochester, USA

DOI: 10.1016/S1474-4422(18)30025-5

read more full text source

rareRelated