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Two brothers battle one fatal disease
05:35 - Source: CNN

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There are only 20,000 cases of Duchenne muscular dystrophy worldwide

The FDA recently approved a controversial drug to treat the condition

CNN  — 

For years, Jenn McNary felt she was fighting a losing battle.

After spending four years petitioning the US Food and Drug Administration to approve the drug eteplirsen, the agency initially advised against it. It was a potential treatment for Duchenne muscular dystrophy, a rare form of the muscle degenerative disease – the form that her two sons have.

Duchenne muscular dystrophy occurs in one out of every 3,600 male infants, and it’s even rarer in girls. The deadly diagnosis is due to an inherited gene mutation, although it can be caused by a random gene mutation, too.

Boys with the condition don’t produce the protein dystrophin, so their muscles are weak and can be easily damaged. Children can appear to develop slowly, and are usually diagnosed by the age of 5. Many with Duchenne rely on wheelchairs as their muscles quickly lose strength, and begin to have difficulty breathing around age 20. Many die from lung disorders within a few years after that.

There is no cure for it, but eteplirsen offered the hope of a longer, better life, and McNary – along with other parents – fought for it.