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Huge breakthrough: This drug fights a terrible disease

February 10, 2017 By Dan Taylor

Huge breakthrough: This drug fights a terrible disease

A major new drug has just gotten approval from the Food and Drug Administration to fight this deadly disease.

It’s an awful disease that destroys the lives of many people who are unfortunate enough to come down with it, and now there is a new drug to fight it. Muscular dystrophy is a group of diseases resulting in progressive weakness and the evaporation of muscle mass over time caused by abnormal genes interfering with the production of proteins needed to form muscle, and the U.S. Food and Drug Administration has approved a new drug meant to combat this process.

It’s called deflazacort, also known under its brand name Emflaza, and the Muscular Dystrophy Association welcomed news of the drug’s approal in a statement. The drug is labeled for use in people who are 5 years of age or older who have Duchenne muscular dystrophy. It’s the third drug in six months approved for drugs in the MDA’s program, the association said.

“Today we celebrate FDA approval of Emflaza as a treatment option for kids and adults living with Duchenne muscular dystrophy,” said MDA President and CEO Steven M. Derks. “This approval follows decades of MDA research — including early-stage development and testing of Emflaza in DMD patients — that was made possible by the hard work, dedication and unwavering commitment of our donors and sponsors. Drugs like Emflaza aren’t developed in silos, but rather through the combined efforts of biotechs and drug companies, patient organizations like MDA, corporate partners and donors, and the families who are there every step of the way to see it through. We are grateful to be part of such a welcome new development in the DMD landscape — and, more importantly, to belong to such a giving and dedicated community.”

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