Dosing & Uses
Duchenne Muscular Dystrophy
Pending FDA approval for Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Pending FDA approval for Duchenne Muscular Dystrophy
Pharmacology
Mechanism of Action
First-in-class steroidal anti-inflammatory drug that differs from conventional glucocorticoids by its lack of an 11β hydroxy-carbonyl group
Removal of this contact site with the glucocorticoid receptor significantly alters structure and activity associations
Additionally, unlike glucocorticoids, vamorolone is not a substrate for 11β-hydroxysteroid dehydrogenase regulatory enzymes and is a mineralocorticoid receptor antagonist
It retains subactivities associated with efficacy (transrepression, physicochemical membrane effects, synchronization of tissue remodeling), and dissociates efficacy from other subactivities associated with detrimental side effects