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L-aminocarnityl-succinyl-leucyl-argininal-diethylacetal
- Development stage
- Preclinical
- Lead developer
- CepTor
- Modality
- Peptide-Drug Conjugates → Peptide Conjugates → Peptides, Small Molecules
01
Overview
L-aminocarnityl-succinyl-leucyl-argininal-diethylacetal (C-101, also known as Myodur) is a synthetic small molecule developed for the treatment of Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy. The compound consists of a carnitine carrier molecule linked to a leupeptin analogue, which acts as an inhibitor of calpain—a protease responsible for skeletal muscle degradation. By inhibiting calpain activity, C‑101 aims to reduce or prevent the accelerated breakdown of skeletal muscle characteristic of these diseases[1][2][3][4].
- Brand names
- Myodur
- Other names
- L-Aminocarnityl-succinyl-leucyl-argininal-diethylacetalUNII-BK727F83NOUNII-BK-727F83NOUNII-BK 727F83NODTXSID90143435DTXSID-90143435DTXSID 90143435
02
Targets
CAPN (Schistosoma calpain large subunit family)
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