Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management.
Duchenne muscular dystrophy (DMD) is a severe, progressive disease that affects 1 in 3600-6000 live male births. Although guidelines are available for various aspects of DMD, comprehensive clinical care recommendations do not exist. The US Centers for Disease Control and Prevention selected 84 clinicians to develop care recommendations using the RAND Corporation-University of California Los Angeles Appropriateness Method. The DMD Care Considerations Working Group evaluated assessments and interventions used in the management of diagnostics, gastroenterology and nutrition, rehabilitation, and neuromuscular, psychosocial, cardiovascular, respiratory, orthopaedic, and surgical aspects of DMD. These recommendations, presented in two parts, are intended for the wide range of practitioners who care for individuals with DMD. They provide a framework for recognising the multisystem primary manifestations and secondary complications of DMD and for providing coordinated multidisciplinary care. In part 1 of this Review, we describe the methods used to generate the recommendations, and the overall perspective on care, pharmacological treatment, and psychosocial management.
Bushby
Katharine
K
Institute of Human Genetics, Newcastle University, Newcastle upon Tyne, UK. kate.bushby@newcastle.ac.uk
Finkel
Richard
R
Birnkrant
David J
DJ
Case
Laura E
LE
Clemens
Paula R
PR
Cripe
Linda
L
Kaul
Ajay
A
Kinnett
Kathi
K
McDonald
Craig
C
Pandya
Shree
S
Poysky
James
J
Shapiro
Frederic
F
Tomezsko
Jean
J
Constantin
Carolyn
C
D003937:Diagnosis, Differential; D005260:Female; D005938:Glucocorticoids / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use; D006801:Humans; D008297:Male; D020388:Muscular Dystrophy, Duchenne / Q000175:diagnosis* / Q000235:genetics / Q000628:therapy*
D005938:Glucocorticoids