Comparative Study of Strategies for Management of Duchenne Myopathy (DM)

Summary

1. Comparing different lines of treatment of Duchenne Myopathy (DM) and assessment of new lines of treatment (mesenchymal stem cell, phosphodiesterase inhibitors) in reducing the impact of disability in the patients with Duchenne Myopathy and slowing the progression of cardiomyopathy 2. Upsetting and implementation of the best treatment plan for those children with Duchenne myopathy which is suitable for the available resources in Assiut University Children Hospital

Timeline

Start

2018-09

Primary completion

2021-09

Completion

2021-11

Publications

• Background Mercuri E, Muntoni F. Muscular dystrophies. Lancet. 2013 Mar 9;381(9869):845-60. doi: 10.1016/S0140-6736(12)61897-2.
• Background Nallamilli BR, Ankala A, Hegde M. Molecular diagnosis of Duchenne muscular dystrophy. Curr Protoc Hum Genet. 2014 Oct 1;83:9.25.1-29. doi: 10.1002/0471142905.hg0925s83.
• Background Lapidos KA, Kakkar R, McNally EM. The dystrophin glycoprotein complex: signaling strength and integrity for the sarcolemma. Circ Res. 2004 Apr 30;94(8):1023-31. doi: 10.1161/01.RES.0000126574.61061.25.
• Background Emery AE. The muscular dystrophies. Lancet. 2002 Feb 23;359(9307):687-95. doi: 10.1016/S0140-6736(02)07815-7.
• Background Wong BL, Christopher C. Corticosteroids in Duchenne muscular dystrophy: a reappraisal. J Child Neurol. 2002 Mar;17(3):183-90. doi: 10.1177/088307380201700306.
• Background Khan MA. Corticosteroid therapy in Duchenne muscular dystrophy. J Neurol Sci. 1993 Dec 1;120(1):8-14. doi: 10.1016/0022-510x(93)90017-s.
• Background Kojima S, Takagi A, Watanabe T. [Effect of prednisolone on apoptosis and cellular infiltration in mdx mouse muscle]. Rinsho Shinkeigaku. 1999 Nov;39(11):1109-13. Japanese.
• Background Fukudome T, Shibuya N, Yoshimura T, Eguchi K. Short-term effects of prednisolone on neuromuscular transmission in the isolated mdx mouse diaphragm. Tohoku J Exp Med. 2000 Nov;192(3):211-7. doi: 10.1620/tjem.192.211.
• Background Wang YX, Rudnicki MA. Satellite cells, the engines of muscle repair. Nat Rev Mol Cell Biol. 2011 Dec 21;13(2):127-33. doi: 10.1038/nrm3265.
• Background Heslop L, Morgan JE, Partridge TA. Evidence for a myogenic stem cell that is exhausted in dystrophic muscle. J Cell Sci. 2000 Jun;113 ( Pt 12):2299-308. doi: 10.1242/jcs.113.12.2299.
• Background Galli R, Borello U, Gritti A, Minasi MG, Bjornson C, Coletta M, Mora M, De Angelis MG, Fiocco R, Cossu G, Vescovi AL. Skeletal myogenic potential of human and mouse neural stem cells. Nat Neurosci. 2000 Oct;3(10):986-91. doi: 10.1038/79924.
• Background Toma JG, Akhavan M, Fernandes KJ, Barnabe-Heider F, Sadikot A, Kaplan DR, Miller FD. Isolation of multipotent adult stem cells from the dermis of mammalian skin. Nat Cell Biol. 2001 Sep;3(9):778-84. doi: 10.1038/ncb0901-778.
• Background Qu-Petersen Z, Deasy B, Jankowski R, Ikezawa M, Cummins J, Pruchnic R, Mytinger J, Cao B, Gates C, Wernig A, Huard J. Identification of a novel population of muscle stem cells in mice: potential for muscle regeneration. J Cell Biol. 2002 May 27;157(5):851-64. doi: 10.1083/jcb.200108150. Epub 2002 May 20.
• Background Shafritz DA, Oertel M, Menthena A, Nierhoff D, Dabeva MD. Liver stem cells and prospects for liver reconstitution by transplanted cells. Hepatology. 2006 Feb;43(2 Suppl 1):S89-98. doi: 10.1002/hep.21047.
• Background Price FD, Kuroda K, Rudnicki MA. Stem cell based therapies to treat muscular dystrophy. Biochim Biophys Acta. 2007 Feb;1772(2):272-83. doi: 10.1016/j.bbadis.2006.08.011. Epub 2006 Sep 6.
• Background Keating A. Mesenchymal stromal cells: new directions. Cell Stem Cell. 2012 Jun 14;10(6):709-716. doi: 10.1016/j.stem.2012.05.015.
• Background Mendell JR, Clark KR. Challenges for gene therapy for muscular dystrophy. Curr Neurol Neurosci Rep. 2006 Jan;6(1):47-56. doi: 10.1007/s11910-996-0009-8.
• Background Partridge TA. Stem cell therapies for neuromuscular diseases. Acta Neurol Belg. 2004 Dec;104(4):141-7.

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Indications