Corticosteroid Deflazacort, Can It Really Cure Muscle Dystrophy?

Currently, there is no approved treatment for the condition called Duchenne muscular dystrophy, which is characterized by muscle weakness that is visible at a young age. This is reinstated with the discovery of corticosteroid deflazacort, which is found to be a promising treatment for the said rare condition.

The study was printed in the journal Neurology. It was led by Robert Griggs, M.D., neurologists from University of Rochester Medical Center (URMC) and colleagues. The findings of the study could lead to the first U.S. approved treatment for muscular dystrophy, according to Medical News Today.

Dr. Griggs explained that Duchenne muscular dystrophy patients have limited treatment options and a desperate need for effective therapies. He further explained that the study indicates that deflazacort may provide a significant treatment for delaying the progression of the disease.

The said drug is approved for use in treating DMD in Europe. Meanwhile, it is not gone through any approval process with the U.S. Food and Drug Administration (FDA).

In the studies conducted by Dr. Griggs and colleagues more than 20 years ago, they showed that the use of corticosteroids every day can heighten the muscle mass and slow muscle degeneration in DMD patients. These enhanced their walking and preserved the patient's respiratory function. On the other hand, several DMD patients in the U.S. are not given corticosteroids because of some concerns about side effects of its prolonged use in children.

Meanwhile, the study involved 196 DMD patients. The results showed that deflazacort was safe and efficient in preserving muscle strength. If deflazacort would be approved, it would be the first drug to treat the said condition in the U.S.

Duchenne muscular dystrophy (DMD) is the type of muscle disease that affects the boys starting at the age of four. This disease could lead to disability with the breakdown of skeletal muscles. Its cause could either be inherited from one's parents or there are mutations in genes that occur in early development. It is reported that there are approximately 28,000 people suffering from the said disease.