“Both my boys deserve to live,” pleads a mother of two boys who suffer from a deadly disease. While 10-year-old Max was approved for a new drug trial, his 13-year-old brother was not. Now, the boys’ mother is pleading with the FDA to help both her boys.
Max and Austin suffer from Duchenne muscular dystrophy, the most common lethal genetic disorder among kids. The disease slowly debilitates the body’s muscles, eventually making it impossible for its victims to walk and, eventually, to breathe. Most patients lost the ability to breathe on their own by the age of 20.
The two Vermont brothers, ages 10 and 13, applied for a trial drug course. Max, 10, was approved by the FDA. Austin, 13, was not. Max has been consistently improving on the drug. Austin, however, is deteriorating.
“If I lose more of my strength I might not be able to hold myself up,” the wheelchair-bound teen says. Of his brother’s improvement since his involvement in the trial, Austin says, “I’m jealous of him, but I’m happy for him, too.”
Austin did not meet the qualifications to receive the trial drug eteplirsen, partly because he is not able to walk.
The brothers‘ parents are pleading with the FDA and asking the drug company to provide the same drug to Austin. Their requests have yet to be approved, and they fear Austin is running out of time.
“I am clearly happy for Max because he is doing so well and exceeding our expectations for this drug,” the boys’ mother, Jenn Mcnary, told ABC News. “But it’s taking much longer than we ever thought for the drug company to at least grant access to his brother [Austin] who is sitting home watching his brother get treatment.”
Sarepta Therapeutics — the company that manufactures the drug — told ABC News that, while the company is sympathetic to Austin’s plight, it’s a “challenging request since it involves complex regulatory, political, manufacturing, and fiscal issues that need to be addressed before a compassionate access program can be developed.”
The drug reportedly works by helping the body produce a protein called dystrophin that is lacking in people who suffer from Duchenne muscular dystrophy. The condition only affects boys, though females can be carriers for the disease. It occurs in one out of every 3,600 male births.
Do you think that both boys should have access to the trial drug?