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vamorolone (Agamree)
Indications:
- treatment of Duchenne muscular dystrophy in patients >= 2 years of age (FDA-approved Oct 2023)
Dosage:
- oral suspension: 2-6 mg/kg/day for up to 48 months.
Adverse effects:
- less effect on bone health, growth trajectory, & behavior than glucocorticoids
Mechanism of action:
- steroidal anti-inflammatory agent
- inhibits proinflammatory NFkB pathways via high-affinity binding to the glucocorticoid receptor
General
anti-inflammatory agent
References
- Brooks M
FDA Okays Drug for Duchenne Muscular Dystrophy
Medscape. October 27, 2023
https://www.medscape.com/viewarticle/997821