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DMD-813

Development stage
Preclinical
Lead developer
DMD Therapeutics
Modality
Small Molecules
01

Overview

**DMD-813** is a proprietary small molecule drug candidate developed by DMD Therapeutics for the treatment of **Duchenne muscular dystrophy (DMD)**, a severe X-linked genetic disorder caused by dystrophin gene mutations leading to progressive muscle degeneration, loss of ambulation, and premature death from cardiorespiratory failure. In preclinical studies using the standard *mdx* mouse model of DMD, DMD-813 demonstrated robust efficacy by reducing muscle inflammation and damage, markedly increasing muscle strength (including in skeletal muscle and diaphragm), improving long-distance ambulation, and reversing key disease effects, positioning it as a promising therapy addressing core pathological mechanisms of muscle weakness in DMD.[1][2][3][4]

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