A genetic condition that causes muscles to waste away has given new hope to patients with Duchenne muscular dystrophy. For an Illinois family, it’s a double blessing after their younger son was diagnosed with the same mutation as their older son.
Duchenne muscular dystrophy is a rare genetic disorder that affects boys and men, causing muscle weakness and wasting. The disease can lead to heart and respiratory failure, and patients often lose the ability to walk by age 10. However, with advancements in gene therapy, doctors are now able to diagnose the condition earlier and offer treatment.
The Flessner family, from rural Illinois, has been through this journey before. Their older son, Mason, received a multi-million dollar dose of synthetic micro genes designed to produce dystrophin, a protein that helps keep muscles intact. After receiving the treatment, Mason made significant progress, including being able to hop and take small steps.
The Flessner boys will help lead the way in this new treatment landscape. Their family has been instrumental in advocating for access to gene therapy, which is now covered by public and private insurances. Two additional gene therapies are currently in development, offering further hope for patients.
“We can’t afford it,” said Daniel Flessner, “but I see what it’s done for our son and I want every child and person affected by it to have access to it.” With the increasing availability of gene therapy, Mason is now able to move around more freely, and his family hopes he will be able to walk normally in the future.
While the treatment is not a cure, doctors hope it will slow down further damage to the body. As Mason continues to receive treatment, the Flessner boys are paving the way for others affected by Duchenne muscular dystrophy.
Source: https://wgntv.com/news/medical-watch/duchenne-muscular-dystrophy-patients-receive-new-hope-with-gene-therapy