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FDA Approves Emflaza Tablet For Duschenne Muscular Dystrophy

Update Date: Feb 13, 2017 08:55 AM EST

The US Food and Drug Administration (FDA) approved on Thursday the Emflaza drug for treatment of Duchenne Muscular Dystrophy (DMD). The drug will be available in oral suspension and tablets. It can be administered to patients 5-years-old and above.

DMD is a rare genetic muscle disorder described by progressive muscle deterioration and weakness. It is caused by lack of protein dystrophin, which helps keep muscles intact.

Emflaza is marketed by Marathon Pharmaceuticals. Boys are greatly affected by DMD, but girls can be mildly affected and be carriers of the gene. DMD affects about one of every 3,600 male infants worldwide according to U.S. News.

There is no cure for DMD only medications that can help improve the patient's way of life. Falls, difficluty getting up from sitting position, difficulty running, a waddling gait, large calves and difficulty learning are symptoms of DMD.

According to CDA News, people with DMD eventually lose ability to carry out activities on their own and are wheelchair bound by the time they become teenagers. Patients develop respiratory and heart related conditions as DMD continues to progress. Patient typically pass away in their 20s or 30s, but life expectancy may vary per patient depending on severity of DMD.

Emplaza is a corticosteroid that reduces activity within the immune system and decreases inflammation. It is the first corticosteroid approved by the FDA that is hopeful to help treat patients with DMD.

Clinical studies of the drug deflazacort showed improvement in muscle strength compare to those who took a placebo. Another study showed patients who took deflazacort lost the ability to walk later than patients who took placebo.

Emflaza side effects are similar to other corticosteroids, such as puffiness of the face, weight gain, increased appetite, infection of the upper respiratory tract, fequent urination, stomach fat, cough, and unwanted hair growth. Other side effects include mood swings, elevated blood pressure, infection and serious skinj rashes.

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