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GRM is an investigational, orally bioavailable small molecule selective glucocorticoid receptor modulator (SGRM) being developed by Grünenthal for the treatment of Duchenne Muscular Dystrophy (DMD). As a dissociative steroid ligand, GRM is designed to separate the therapeutic anti-inflammatory and muscle-protective effects of glucocorticoids from the debilitating side effects typically associated with chronic corticosteroid use, such as bone loss, growth suppression, and metabolic dysfunction. The molecule works by selectively binding to the glucocorticoid receptor to favor transrepression pathways—which inhibit pro-inflammatory transcription factors like NF-κB—while minimizing the transactivation of genes responsible for systemic adverse effects. GRM is currently in early-stage clinical development to evaluate its safety, tolerability, and pharmacokinetics in humans.
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