Help boys with DMD preserve their
"here and now"
As the first and only FDA‑approved corticosteroid for boys with DMD as young as age 2, EMFLAZA is designed to help maintain muscle strength and function.
In phase 1 (from baseline to 12 weeks) of a 52‑week clinical trial with 196 boys aged 5 to 15 years with DMD, the effectiveness and safety of EMFLAZA were compared to placebo (sugar pill). After 12 weeks of treatment, patients taking EMFLAZA had significantly improved muscle strength compared with placebo (0.15 change in strength score vs -0.10 change in strength score).
Make the Most
of His Now
Help delay dmd progression,
When it matters
In a 10-year follow-up study measuring 3 important DMD events, deflazacort showed differences over time compared to prednisone by helping boys maintain:
In a study of 440 boys aged 2 to 28 years with DMD, the effectiveness and safety of deflazacort were compared with prednisone.
Age at loss of ambulation with deflazacort vs prednisone
Age at Loss of ambulation
Bello 2015 Study Summary
Prednisone/prednisolone and deflazacort regimens in CINRG Duchenne Natural History Study
Objective: This study observed the age at loss of independent ambulation (LoA) and side-effect profiles associated with different glucocorticoid corticosteroid (GC) regimens in DMD.
Methods: This observational study assessed 340 participants aged 2 to 28 years in the Cooperative International Neuromuscular Research Group Duchenne Natural History Study (CINRG-DNHS). LoA was defined as continuous wheelchair use. Effects of prednisone or prednisolone/deflazacort, administration frequency, and dose were analyzed by time-varying Cox regression (a method to estimate survival time). Side-effect frequencies were compared using x2 test.
Results: Participants treated 1 year or longer while ambulatory showed a 3-year median delay in LoA. Fourteen different regimens were observed. Non-daily treatment was common for prednisone (37%) and rare for deflazacort (3%). Deflazacort was associated with later LoA than prednisone. Average dose was lower for daily prednisone (75% of the recommended dose) than daily deflazacort (83% of the recommended dose). Deflazacort showed higher frequencies of growth delay, cushingoid appearance, and cataracts, but not weight gain.
This study is not in the approved prescribing label for EMFLAZA, but is consistent with the information that is included. Please talk to your son's healthcare provider if you have any questions.
Help support him
as he grows
Once your son starts taking deflazacort, it's important for him to stay on treatment as it was shown to preserve muscle function in more places than just his arms and legs.
In a 13-year study, scoliosis was delayed and lung function was preserved for longer in boys taking deflazacort.
A 13-year study of 435 boys with DMD assessed patients' ages simultaneously with DMD progression events, including scoliosis onset and decrease in lung function.
Here & now with
Sarah and Dan work together to care for their two sons with DMD. Watch how they try to have fun and enjoy life as much as they can.Emflaza Safety Profile