Drug intelligence / Profile preview

deflazacort + prednisone

Development stage
Unknown
Lead developer
Marathon Pharmaceuticals
Modality
Orthosteric Ligands → Classical Binding Small Molecules → Small Molecules
Administration
Oral
01

Overview

Deflazacort and prednisone are synthetic glucocorticoids used in the treatment of Duchenne muscular dystrophy (DMD). Both medications help improve muscle strength, delay loss of ambulation, improve pulmonary function, reduce the need for scoliosis surgery, delay onset of cardiomyopathy, and increase survival in DMD patients. They differ in their pharmacological properties, with deflazacort having lower lipid solubility, causing less sodium retention, and having fewer effects on glucose metabolism compared to prednisone. Evidence suggests that deflazacort may be more effective than prednisone at delaying disease progression in DMD, particularly in terms of muscle strength, motor function, and time to loss of ambulation. Regarding side effects, weight gain and behavioral issues appear to be greater with prednisone, whereas bone health issues, growth parameters, and cataracts appear worse with deflazacort.

02

Targets

GR (Glucocorticoid receptor)

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