Update on the Treatment of Duchenne Muscular Dystrophy
Author:
Publisher
Springer Science and Business Media LLC
Subject
Neurology (clinical),General Neuroscience
Link
http://link.springer.com/content/pdf/10.1007/s11910-012-0332-1.pdf
Reference62 articles.
1. •• Mendell JR, et al. Evidence-based path to newborn screening for Duchenne muscular dystrophy. Ann Neurol. 2012;71:304–13. This was the first report of a two-tier system using dried blood spots obtained at birth to identify cases of DMD. Creatine kinase levels are first determined and if they reach a level predictive of DMD, DNA sequencing follows.
2. Koenig M, et al. Complete cloning of the Duchenne muscular dystrophy (DMD) cDNA and preliminary genomic organization of the DMD gene in normal and affected individuals. Cell. 1987;50:509–17.
3. Brooke MH, et al. Clinical investigation in Duchenne dystrophy: 2. Determination of the “power” of therapeutic trials based on the natural history. Muscle Nerve. 1983;6:91–103.
4. England SB, et al. Very mild muscular dystrophy associated with the deletion of 46 % of dystrophin. Nature. 1990;343:180–2.
5. Harper SQ, Hauser MA, DelloRusso C, et al. Modular flexibility of dystrophin: Implications for gene therapy of Duchenne muscular dystrophy. Nat Med. 2002;8:253–61.
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