DEFLAZACORT (deflazacort) by PTC Therapeutics is prodrug, whose active metabolite, 21-desdfz, acts through the glucocorticoid receptor to exert anti-inflammatory and immunosuppressive effects. Approved for duchenne muscular dystrophy (dmd) in patients 5 years of age, older ( ). First approved in 2025.
Drug data last refreshed 21h ago
prodrug, whose active metabolite, 21-desDFZ, acts through the glucocorticoid receptor to exert anti-inflammatory and immunosuppressive effects. The precise mechanism by which deflazacort exerts its therapeutic effects in patients with DMD is unknown.
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A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)
A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)
Food Effect and Bioavailability of Deflazacort Formulations in Healthy Volunteers
A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy
An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort