Christine McSherry is used to a fight. Her son, Jett, was diagnosed with Duchenne muscular dystrophy, a rare, debilitating condition, when he was 5. He lost the ability to walk just before he turned 14.
McSherry has become an outspoken advocate for Jett and other kids with Duchenne, particularly in fighting for access to drugs she's convinced help against the disease's devastating effects, CNBC reported.
Jett, now 21, has been taking a steroid called deflazacort since he was 8. Deflazacort wasn't approved in the U.S., so McSherry and a number of other families of kids with Duchenne imported the drug from overseas. She says it cost about $1,200 a year out of pocket.
On Monday, deflazacort got a new price tag: $35,000 a year for a patient in the U.S. weighing 25 kilograms, or about 55 pounds.
PTC Therapeutics, which set the price, says it "represents sustainable pricing which balances providing access to eligible patients in the United States…while maintaining sufficient infrastructure and programs including continued investment in Duchenne."