PHILADELPHIA, Pa. (Ivanhoe Newswire) – Once a death sentence, hemophilia now has a cure. The genetic disorder primarily affects boys and occurs when the blood does not clot. Hemophilia can cause spontaneous severe bleeding after an injury. Twenty years ago, the only treatment available was to stop the bleeding. Now, gene therapy has further increased the possibility of one day curing the disease.
The Ward brothers may have different interests, but Jadon and Lorne have more in common than meets the eye.
“Hemophilia is a condition where there is excessive bleeding,” Roan says.
“My blood doesn’t clot like a normal human’s,” Jadon explained.
Both were born with hemophilia.
“Bleeding is definitely their biggest threat,” said their mother, Melody Ward.
“When I started treating people 50 years ago, most people with hemophilia had some kind of deformity or disability. You know, some in wheelchairs, some in braces, some on crutches. “Some people had this problem,” explained Regina Butler, M.D., clinical manager of hematology at Children’s Hospital of Philadelphia.
Butler has treated five generations of Melody’s family, including Melody’s father, uncle and now her sons, who receive injections every few weeks to prevent bleeding.
“Therapies are evolving rapidly. We kept coming up with better and better products,” Butler said.
A new gene therapy for hemophilia B has been approved by the FDA. With a single infusion, Hemgenics instructs the body to produce a functional factor 9 gene, which helps blood clot.
“That’s remarkable to me. I feel like my hemophilia career has come full circle,” Butler said.
Hemgenics can only be administered once, but its effects can last for years, making the injections the boys in the ward receive obsolete.
“I never thought I’d see the day when I could say there was a cure for hemophilia,” Melody said.
The treatment is currently approved for patients 18 and older, but doctors hope to extend the treatment to children as young as 12 in the future. The most common side effects associated with Hemgenics include elevated liver enzymes, headaches, and flu-like symptoms.
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