1006685-44-4CCOC(OCC)[C@H](CCCN=C(N)N)NC(=O)[C@H](CC(C)C)NC(=O)CCC(=O)N[C@H](CC(=O)[O-])C[N+](C)(C)CJOITUBWXBFATOR-HKBOAZHASA-NC-101, also called Myodur, is developed for the treatment of Duchenne’s muscular dystrophy (DMD) which is a morbid genetic disease that can lead to death in late adolescence due to accelerated skeletal muscle breakdown. C-101 includes a carnitine carrier molecule and a leupeptin analogue, a known calpain inhibitor. Calpain is the primary protease that degrades skeletal muscle.
1006685-44-4DTXSID90143435C-101 targets muscle cells by using a carnitine carrier molecule and inhibits calpain by attaching a leupeptin analogue onto the carrier. Leupeptin has been studied extensively in a variety of animal models and was shown to be an effective means of slowing or delaying muscle wasting. Carnitine is a compound present in skeletal muscle involved in the transfer of fatty acids across mitochondrial membranes. In C-101, the carnitine carrier molecule directs the leupeptin analogue into targeted cells where it inhibits the up-regulation of calpain. Therefore, by targeting a calpain inhibitor to muscle cells, it is believed that C-101 will prevent the degradation and promote the preservation of functional muscle.
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