L-aminocarnityl-succinyl-leucyl-argininal-diethylacetal
L-aminocarnityl-succinyl-leucyl-argininal-diethylacetal Uses, Dosage, Side Effects, Food Interaction and all others data.
C-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.
Table Of contents
| Trade Name | L-aminocarnityl-succinyl-leucyl-argininal-diethylacetal |
| Generic | L-aminocarnityl-succinyl-leucyl-argininal-diethylacetal |
| Type | |
| Formula | C27H53N7O7 |
| Weight | Average: 587.763 Monoisotopic: 587.400647075 |
| Groups | Investigational |
| Therapeutic Class | |
| Manufacturer | |
| Available Country | |
| Last Updated: | September 19, 2023 at 7:00 am |
Uses
Investigated for use/treatment in muscular dystrophy.
How L-aminocarnityl-succinyl-leucyl-argininal-diethylacetal works
C-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.
Innovators Monograph
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