FDA approves Emflaza to treat Duchenne muscular dystrophy

What is Emflaza?

Emflaza (Deflazacort) is a glucocorticoid, a class of corticosteroids used as an anti-inflammatory and immunosuppressant. Deflazacort works by decreasing inflammation and reducing the activity of the immune system to treat certain conditions such as: acute interstitial nephritis, anaphylaxis, asthma, anemia, Crohn’s disease, dermatomyositis, idiopathic thrombocytopenic purpura, juvenile chronic arthritis, acute and lymphatic leukemia, malignant lymphoma, rheumatoid arthritis, nephrotic syndrome, stemic lupus erythematosus, ulcerative colitis and many others.

What is Duchenne Muscular Dystrophy?

Duchenne muscular dystrophy is a rare genetic condition that weakens and deteriorates the muscles. The symptom of muscle weakness usually begins around 3 to 5 years, specially boys, affecting between 3,500 and 5,000 males at birth worldwide. Duchenne muscular dystrophy occurs due to an absence of dystrophin, a protein that protects muscle cells membranes. Two thirds of cases the patients inherited it from the parents, while one third of cases are due to a new mutation that affects the protein.

Emfalza (Deflazacort) works by improving muscle strength in patients with Duchenne muscular dystrophy, and it comes tablets and oral suspension.