Title: Life-Altering Gene Therapy Treatment for Duchenne Muscular Dystrophy Approved by FDA
Subtitle: Norfolk 5-year-old becomes part of “historic” gene therapy event at CHKD
May 4, 2023 – Norfolk, Virginia: The parents of a 5-year-old boy with Duchenne Muscular Dystrophy (DMD) are filled with hope as their child undergoes a groundbreaking gene therapy treatment at Children’s Hospital of The King’s Daughters (CHKD). In June, the Food and Drug Administration (FDA) approved a gene therapy treatment that doctors claim is life-altering for patients with DMD.
Karson Blanchard, a lively kindergartener who loves mac and cheese and chocolate, has spent a significant portion of his young life in and out of hospital rooms. Karson’s mother, Katy Blanchard, explained that Karson did not hit his gross motor milestones during infancy, leading doctors to diagnose him with Duchenne Muscular Dystrophy, a rare genetic condition that causes progressive wasting of the muscles.
DMD is typically recognized in children between the ages of three and six and often shortens their lifespans. Many patients with DMD experience heart and respiratory failure in their 20s and 30s. While Karson has received some treatment for the condition, he arrived at CHKD in Norfolk to try the new gene therapy called Elevidys.
Katy Blanchard expressed her excitement about the possibilities this treatment offers for her son’s future. She said, “It really opens a lot of doors for what-ifs that weren’t possible before. Of course, we don’t know what exactly it will look like, but now we can wonder whether he will have to get therapy every week forever, or maybe he’ll be able to walk through high school or into adulthood, where that really wasn’t a possibility before.”
The gene therapy treatment involves introducing a protein similar to the one missing in Karson’s muscles. While it cannot reverse muscle damage, doctors believe it has the potential to prevent future impairments. CHKD is the first hospital in Virginia to administer this therapy and plans to extend it to other eligible patients in the near future.
One of the significant benefits for Karson, according to his own words, is “not having to get poked.” However, the treatment is currently limited to patients between the ages of four and five, while Karson will turn six in a few days. Despite this, doctors remain hopeful about the positive outcomes of the gene therapy.
The approval and availability of the gene therapy treatment represent a significant breakthrough for patients with Duchenne Muscular Dystrophy, offering renewed hope to families like the Blanchards. As medical advancements continue to transform the landscape of rare genetic conditions, the future looks brighter for children like Karson, allowing them to dream of a life beyond the limitations imposed by their condition.