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AGAMREE (vamorolone) is a novel corticosteroid approved for Duchenne muscular dystrophy (DMD), designed to modulate inflammation while potentially mitigating traditional steroid-related adverse effects. It binds to the glucocorticoid receptor, exerting anti-inflammatory and immunosuppressive actions, though its precise mechanism in DMD remains under study.
Unlike conventional corticosteroids, vamorolone demonstrates dissociative properties that may preserve bone health and growth metrics, as suggested by preclinical models. Clinical trials highlight its efficacy in improving functional endpoints such as Time to Stand Test (TTSTAND) velocity and 6-Minute Walk Test (6MWT) distance, with a safety profile characterized by lower incidence of cushingoid features compared to prednisone.
AGAMREE is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older.
Initial Dose: 6 mg/kg orally once daily with food (max 300 mg/day for patients >50 kg).
