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Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study
Craig M McDonald ,
Erik K Henricson ,
Richard T Abresch ,
Tina Duong ,
Nanette C Joyce ,
Fengming Hu ,
Paula R Clemens ,
Eric P Hoffman ,
Avital Cnaan ,
Heather Gordish-Dressman ,
Vijay Vishwanathan ,
S Chidambaranathan ,
W. Douglas Biggar ,
Laura C. McAdam ,
Jean K. Mah ,
Mar Tulinius ,
Avital Cnaan ,
Lauren P. Morgenroth ,
Robert Leshner ,
Carolina Tesi-Rocha ,
Mathula Thangarajh ,
Tina Duong ,
Andrew Kornberg ,
Monique Ryan ,
Yoram Nevo ,
Alberto Dubrovsky ,
Paula R. Clemens ,
Hoda Abdel-Hamid ,
Anne M. Connolly ,
Alan Pestronk ,
Jean Teasley ,
Tulio E. Bertorin ,
Richard Webster ,
Hanna Kolski ,
Nancy Kuntz ,
Sherilyn Driscoll ,
John B. Bodensteiner ,
Jose Carlo ,
Ksenija Gorni ,
Timothy Lotze ,
John W. Day ,
Peter Karachunski ,
Erik K. Henricson ,
Richard T. Abresch ,
Nanette C. Joyce ,
Craig M. McDonald
February 2018
February 2018
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Abstract
Glucocorticoid treatment is recommended as a standard of care in Duchenne muscular
dystrophy; however, few studies have assessed the long-term benefits of this treatment.
We examined the long-term effects of glucocorticoids on milestone-related disease
progression across the lifespan and survival in patients with Duchenne muscular dystrophy.