US FDA approved new medicine Emflaza that contains active ingredient deflazacort in oral dosage form for the treatment of Duchenne muscular dystrophy (DMD). Emflaza belongs to a therapeutic class termed as corticosteroids. Emflaza is basically a prodrug which is converted in active metabolite after its metabolism. All corticosteroids works by reducing swelling, inflammation and settling down the exaggerated immune response.
Duchenne muscular dystrophy (DMD) is a rare kind of genetic disorder that causes degeneration of muscles. Due to some genetic defect a protein termed as dystrophin does not produce in the body. The basic physiological role of dystrophin is keeping body muscles intact and healthy.
But dystrophin absence in Duchenne muscular dystrophy (DMD) makes muscle more vulnerable to degradation, degeneration and physical deterioration. Symptoms may first appear in the age of 5 years or earlier. After the onset of disease symptoms may get worst with the passage of time.
In extreme cases duchenne muscular dystrophy (DMD) may cause walking disability, cardiovascular or respiratory muscles collapse. These disabilities may appear at any time without regard to age. According to statistics this disease is more common in boys as compared to girls.
Emflaza/deflazacort Side effectsAccording to clinical data provided by health authorities Emflaza demonstarted a significant results over placebo. However, side effects were similar to other corticosteroids therapy. Most commonly observed side effects were as follows
- Weight gain
- Hair growth (hirsutism)
- Central obesity
- Facial puffiness (Cushingoid appearance)
- Upper respiratory tract infection
- Increased appetite
- Urinary frequency