The good news for Manalapan 6-year-old Jamesy Raffone: He’s way ahead of his peers in battling Duchenne muscular dystrophy.
The bad news: He’s too far ahead to qualify for the latest clinical trial.
That was the verdict from Cincinnati Children’s Hospital Medical Center, which is pioneering treatment for the rare and fatal disease. Jamesy, whose cause has been taken up by thousands of people in Central Jersey over the past year, traveled there for a series of tests last month.
“He is three seconds faster than (his peers) in the 10-meter run,” his father, Jim Raffone, said. “He’s 1.3 seconds faster than any other boy his age getting off the ground unassisted from sitting Indian-style. They won’t put him in the trial because they said he’s too strong. They would give the spot to another child who is not as strong.”
The Raffones take consolation in how well Jamesy is doing since his diagnosis two years ago. Duchenne is a genetic condition that results in a progressive loss of strength due to the absence of a critical muscle-building protein. Eventually the weakness leads to paralysis and fatal heart and lung problems. Life expectancy is in the 20s.
Because Jamesy’s strain of Duchenne is so rare — there are fewer than 200 boys with the condition in the United States — research and treatment options were scarce. So the family started their own foundation, JAR of Hope, and it has raised more than $150,000 thanks to a tremendous groundswell of local support.