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FDA Approves Emflaza For Treating Duchene Muscular Dystrophy

By jeremyc | February 10, 2017

The FDA has approved Emflaza (deflazacort) for treatment of Duchene muscular dystrophy in people age 5 years and older. Duchene muscular dystrophy (also known as DMD) is genetic disorder in which muscles progressively become weak and deteriorate. DMD is a rare but fatal condition. At present, it is believed that nearly 15,000 individuals in the U.S. are living with this condition. Emflaza is available in form of oral tablets as well as oral suspension.

Emflaza belongs to the category of corticosteroids, which work by suppressing inflammation. Corticosteroids are frequently used for treating DMD in many countries. Emflaza is the first corticosteroid to get approval for treating DMD in the U.S. The disease occurs because of lack of dystrophin, an important protein responsible for keeping muscles strong and healthy.

DMD is more common than any other muscular dystrophy, a disease that causes muscles to become progressively weaker and less flexible. This condition mainly affects boys. The disease symptoms start appearing early in childhood. DMD affected children have trouble standing up and walking. Many children eventually require a wheelchair. Lung and heart problems are also common in children who’ve DMD.

Like other corticosteroids, DMD can cause certain side effects, like facial puffiness, increased appetite, weight gain, and infection in the upper respiratory tract, among others.

 

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