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|Year : 2018 | Volume
| Issue : 2 | Page : 582-583
Author's Reply: Standard of care for Duchenne muscular dystrophy in children
Department of Neurology, National Institute of Mental Health and Neurosciences, Bengaluru, Karnataka, India
|Date of Web Publication||15-Mar-2018|
Dr. A Nalini
Department of Neurology, National Institute of Mental Health and Neurosciences, Bengaluru, Karnataka
Source of Support: None, Conflict of Interest: None
|How to cite this article:|
Nalini A. Author's Reply: Standard of care for Duchenne muscular dystrophy in children. Neurol India 2018;66:582-3
In our article, we have clearly compared the outcome among those who have received steroids vs. those who have not. Further, treatment with steroids is a standard of care for children with Duchenne muscular dystrophy (DMD) for the last few decades and it would be impossible to perform a natural history study on drug naïve children. There are earlier studies on the natural history where both groups have been included for outcome studies. In the study by Roberto et al., 174 male patients with DMD, ranging from 9 to 35 years, with an average age at the initial visit of almost 21 years were studied. 72 subjects were on steroids, 22 subjects were not on steroids, and the steroid treatment status of 80 subjects was unknown. In another study by Bushby et al., the tests conducted best demonstrated differences in steroid-treated and naive populations from the youngest ages into the mid-teens, and were most sensitive to 1-year changes between 7–9 and 10–12 years of age for standing from supine, and 7–9, 10– 12 and 13–18 years for stair climb and 10-m run/walk.
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| » References|| |
Singh RJ, Manjunath M, Preethish-Kumar V, Polavarapu K, Vengalil S, Thomas PT, et al
. Natural history of a cohort of Duchenne muscular dystrophy children seen between 1998 and 2014: An observational study from South India. Neurol India 2018;66:77-82.
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Roberto R, Fritz A, Hagar Y, Boice B, Skalsky A, Hwang H, et al
. The natural history of cardiac and pulmonary function decline in patients with Duchenne muscular dystrophy. Spine 2011;36:E1009-E1017.
Bushby K, Connor E. Clinical outcome measures for trials in Duchenne muscular dystrophy: report from International Working Group meetings. Clinical investigation 2011;1:1217-35.