Emflaza (Deflazacort) for Muscular dystrophy

Overview
Emflaza is approved by the U.S. Food and Drug Administration (FDA) for the treatment of Duchenne muscular dystrophy (DMD) in people 2 years of age and older. It is specifically indicated for use in children and adults with DMD, a genetic disorder characterized by progressive muscle degeneration and weakness. Emflaza is also known by its drug name, deflazacort.

Deflazacort is a corticosteroid, a type of medication that reduces inflammation and suppresses the immune system. For DMD, deflazacort helps slow the progression of muscle weakness, although it is not a cure for the condition.

How do I take it?
Prescribing information states that Emflaza should be taken orally once daily, with dosing based on the recipient’s weight. It is available in both tablet and oral suspension forms to accommodate different needs. The medication should be discontinued gradually if taken for more than a few days to prevent potential withdrawal effects. Emflaza should be taken exactly as prescribed by a health care provider.

Side effects
Common side effects of Emflaza include cushingoid appearance (rounded face), weight gain, increased appetite, upper respiratory tract infections, cough, frequent urination, increased body hair, central obesity, and nasal inflammation or sore throat.

Rare but serious side effects may include suppression of adrenal gland function, high blood sugar, increased blood pressure, electrolyte imbalances (such as high sodium and low potassium levels), gastrointestinal perforation (tear in the stomach or intestines), behavioral or mood changes, bone loss, cataracts, glaucoma (increased eye pressure), and severe skin rashes. Additionally, Emflaza can increase the risk of infections, and live vaccines should not be given while taking it.

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Label: Emflaza — Deflazacort Tablet, Emflaza — Deflazacort Suspension — DailyMed