People with the bleeding disease hemophilia now have a breakthrough treatment option developed from research at Children’s Hospital of Philadelphia.<\/p>\n
The gene therapy works with one infusion. It doesn’t cure hemophilia, but it makes it a whole lot safer and easier.<\/p>\n
Domenic Catrine doesn’t have to worry anymore about getting scratched while playing with his dog.<\/p>\n
“I’m extremely blessed and grateful,” Catrine said.\u00a0<\/p>\n
The South Jersey college student was born with hemophilia B \u2013 a rare condition that can cause dangerous bleeding because the blood doesn’t clot properly.<\/p>\n
“You never know what can happen, what can cause a bleed,” Catrine said. “It can be a little scary living with hemophilia.”<\/p>\n
As a little boy, Catrine wore a helmet to reduce the risk of bleeds and also had to get regular infusions to control the condition.<\/p>\n
“Poking myself every week was not something I was very happy with doing,” he said.\u00a0<\/p>\n
Now, the 21-year-old doesn’t have to rely on infusions. Catrine is the first patient to get a new Food and Drug Administration-approved gene therapy called Hemgenix.<\/p>\n
“It’s put me in a better position and a better position to live life more freely,” Cartine said.\u00a0<\/p>\n
Research at CHOP, where Catrine was treated, was instrumental in developing the gene therapy.<\/p>\n
“I think this is a huge development, but it’s been three decades in the making,” Dr. Ben Samelson-Jones, a CHOP hematologist, said.\u00a0<\/p>\n
Samelson-Jones said patients like Catrine have a missing gene that causes the bleeding disorder. The gene therapy replaces it.<\/p>\n
“What it does is certainly converts patients from what we call the severe disease, where you’re at risk for having spontaneous, potentially life-threatening bleeds to a much more mild disease, where you would only need additional treatment if you had major trauma or surgery,” Samelson-Jones said.\u00a0<\/p>\n
Catrine said it was an easy one-time injection, with no side effects. Now, he doesn’t have to worry about injuries or getting regular infusions.<\/p>\n
“I never imagined a world where I wouldn’t have to infuse in some way,” Cartine said. “That was mind-blowing to me.”<\/p>\n
Catrine isn’t cured but now has what he calls a more normal life.<\/p>\n","protected":false},"excerpt":{"rendered":"People with the bleeding disease hemophilia now have a breakthrough treatment option developed from research at Children’s Hospital…\n","protected":false},"author":2,"featured_media":58636,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[8],"tags":[32056,69,71,70],"class_list":{"0":"post-58635","1":"post","2":"type-post","3":"status-publish","4":"format-standard","5":"has-post-thumbnail","7":"category-philadelphia","8":"tag-children039s-hospital-of-philadelphia","9":"tag-philadelphia","10":"tag-philadelphia-headlines","11":"tag-philadelphia-news"},"_links":{"self":[{"href":"https:\/\/www.newsbeep.com\/us-pa\/wp-json\/wp\/v2\/posts\/58635","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.newsbeep.com\/us-pa\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.newsbeep.com\/us-pa\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/us-pa\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/us-pa\/wp-json\/wp\/v2\/comments?post=58635"}],"version-history":[{"count":0,"href":"https:\/\/www.newsbeep.com\/us-pa\/wp-json\/wp\/v2\/posts\/58635\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/us-pa\/wp-json\/wp\/v2\/media\/58636"}],"wp:attachment":[{"href":"https:\/\/www.newsbeep.com\/us-pa\/wp-json\/wp\/v2\/media?parent=58635"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.newsbeep.com\/us-pa\/wp-json\/wp\/v2\/categories?post=58635"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.newsbeep.com\/us-pa\/wp-json\/wp\/v2\/tags?post=58635"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}