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NRDN-301
- Development stage
- Preclinical
- Lead developer
- Neurodon
- Modality
- Small Molecules
01
Overview
NRDN-301 is a small molecule activator of the sarco/endoplasmic reticulum Ca2+-ATPase (SERCA) enzyme, currently in preclinical development by Neurodon for the treatment of Duchenne muscular dystrophy (DMD). In DMD, calcium dysregulation in muscle cells leads to muscle weakening, as well as cardiac and respiratory dysfunction. NRDN-301 binds to an allosteric site on SERCA to accelerate its activity, thereby restoring cellular calcium homeostasis and potentially mitigating muscle cell damage. This approach represents a novel therapeutic strategy for DMD by addressing the underlying calcium imbalance implicated in the disease's progression.
02
Targets
ATP2A (Sarcoplasmic reticulum Ca2+-ATPase)
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