WEST LONG BRANCH, N.J.–(BUSINESS WIRE)–JAR of Hope (https://jarofhope.org), a foundation researching a cure for a fatal children’s disease called Duchenne muscular dystrophy, has just received good news. The Food & Drug Administration has approved an IND (Investigational New Drug) application to begin clinical trials of a compound called JAR914. Since September of 2019, JAR of Hope helped to fund this new IND.
Duchenne muscular dystrophy is a very rare, muscle-wasting disease with which children are born…only 1 in every 3,500 live births. By the age of 11 or 12, these kids—generally boys—can start losing the ability to walk. By the early-teens, they’re in wheelchairs. By the late teens, on ventilators. And by the early twenties, in graves. From suffocation.
“We’re fighting to save the lives of children with a disease for which there’s currently no cure,” says Jim Raffone, CEO/Founder of JAR of Hope. “This FDA approval will allow us to continue our efforts to find a cure with our ‘Operation: LifeLine’ initiative.”
Jim and Karen Raffone started JAR of Hope after their own son, James Anthony, now 11, was diagnosed with Duchenne in 2013.
Some members of the scientific community believe JAR914 can possibly extend the lives of children with Duchenne. JAR of Hope has permission to involve up to 12 children in these clinical studies, which will focus on the safety of the children and the efficacy of the compound.
Only 20,000 children in the US have Duchenne. Statistically, that means perhaps 500–600 live in New Jersey. And perhaps twice that number in New York State.
“When the doctors told Karen and I to ‘just take your son home and love him until he dies,’ Jim Raffone says, “that just didn’t sit well with us. So we decided to fight for all the families facing this horrible disease. It’s an expensive fight…it will take about $2 million just to get started with these clinical trials. But this is one fight we’re determined to see through…all the way to the finish.”
About JAR of Hope:
JAR of Hope was founded in 2013 by James Raffone, after his young son, Jamesy, was diagnosed with Duchenne Muscular Dystrophy. Duchenne is a form of muscular dystrophy that is caused by a mutation in the dystrophin gene. The absence of dystrophin results in muscle deterioration – leading to paralysis, decreased cardiac function, and eventual death. Duchenne affects about 300,000 young males worldwide with approximately 20,000 cases in the United States. Currently, Duchenne has no cure and the average life expectancy of those affected is 25 years. However, JAR of Hope is devoted to eliminating the disease by bringing awareness and raising funds directed to ongoing research.