Emflaza (deflazacort)

The first corticosteroid specifically FDA-approved for DMD. Slows muscle deterioration and helps boys maintain strength and motor function longer than without treatment.

Deflazacort calms the immune system's inflammatory attack on damaged muscle fibers. In DMD, muscles break down with every contraction and the body's repair process triggers chronic inflammation that causes further damage. By reducing this inflammation, deflazacort slows the cycle of muscle destruction and helps preserve strength.

Weight gain, increased appetite, upper respiratory infections, frequent urination, and cushingoid appearance are common. Long-term corticosteroid use can cause bone thinning, cataracts, growth suppression, and mood changes. Requires monitoring of bone density and blood sugar. A rare but life-threatening skin reaction called toxic epidermal necrolysis has been reported, with symptoms starting within 8 weeks of starting treatment, so call the doctor at the first sign of a rash. Steroids also raise the risk of infections, which can be serious or even fatal, and live vaccines should be given at least 4 to 6 weeks before starting, not during treatment. The label also warns about high blood pressure and low potassium, tears in the stomach or intestines, glaucoma and eye infections, a muscle problem called myopathy, blood clots, a rare cancer called Kaposi sarcoma, and rare severe allergic reactions (anaphylaxis).

Taken by mouth once daily as tablets or oral suspension. Typical dose is 0.9 mg/kg/day. Approved for patients age 2 and older.

Yes, Deflazacort (Emflaza) is FDA approved (2017) for the treatment of Duchenne Muscular Dystrophy.

Both are corticosteroids that slow muscle deterioration in DMD, and both are considered standard of care. Clinical data suggests they have similar efficacy for preserving motor function and extending ambulation. The key difference is side effects: deflazacort tends to cause less weight gain than prednisone, which is important for boys who already have difficulty with mobility. However, deflazacort may cause slightly more risk of cataracts. Many DMD care teams now prefer vamorolone (Agamree) as a newer option with fewer steroid side effects.

Yes. Generic deflazacort became available after patent protections ended, significantly reducing costs. Generic versions are available from multiple manufacturers and contain the same active ingredient at the same doses. The availability of generics makes deflazacort one of the more affordable DMD treatment options.

Long-term corticosteroid use in DMD commonly causes weight gain, cushingoid appearance, bone thinning (osteoporosis), growth suppression, cataracts, increased infection risk, mood and behavioral changes, and adrenal suppression. Bone density monitoring (DEXA scans) is recommended, and calcium and vitamin D supplementation is standard. These side effects are a major quality-of-life concern for DMD families and one reason newer alternatives like vamorolone are being developed.

Current DMD care guidelines recommend starting corticosteroid therapy when motor function has plateaued or is beginning to decline, typically between ages 4 and 6. Starting too early may cause unnecessary side effects, while starting too late may miss the window for maximum benefit. The exact timing should be individualized based on the child's functional status and discussed with a neuromuscular specialist.

Deflazacort should not be stopped abruptly because long-term corticosteroid use suppresses the adrenal glands' ability to produce cortisol naturally. Abrupt discontinuation can cause an adrenal crisis, which is a medical emergency. If stopping or reducing the dose is necessary, it must be done gradually under medical supervision. Most DMD care teams recommend continuing corticosteroids throughout the disease course.