How well does Emflaza(Deflazacort) work?

This section summarizes the clinical evidence supporting the drug's effectiveness.

Clinical Effectiveness

The efficacy of EMFLAZA for DMD was demonstrated in a 52-week, placebo-controlled clinical trial. The primary endpoint was the change in average muscle strength score. Patients treated with deflazacort 0.9 mg/kg/day showed a statistically significant improvement in muscle strength compared to those on placebo at week 12. This treatment effect was observed to persist over time, supporting its use for maintaining muscle function in patients with DMD.