US EditionRocky Ford Family Raising Money for Possible Cure to Son’s Rare, Terminal Disease
- Experimental gene therapy for Fabry disease is saving treatment costs for patients, as reported by West.
- Three men treated for Fabry disease discontinued enzyme-replacement therapy, which costs about $300,000 annually, after starting gene therapy.
- Larger studies are needed before gene therapy can become a conventional treatment due to existing therapy's high costs and patient burden.
- The goal is to expand the study to include 25 to 30 patients over a two- to three-year period, according to West.
23 Articles
23 Articles
Rocky Ford family raising money for possible cure to son’s rare, terminal disease
COLORADO SPRINGS, Colo. (KRDO) - A Rocky Ford family is sharing their story as they fight to save their young son’s life. Three-year-old Mason Matthews has a rare and terminal genetic condition called Mucolipidosis Type II, something most people have never heard of. Mason’s mom, Jaymie Matthews, said they first knew something was wrong at birth when their son was born full term, but couldn’t breathe on his own. "He had a very traumatic birth exp…
Ariel was born with a serious genetic disease, her father Dr. Shternin decided not to give up and find a cure • At the end of the journey, he managed to reach a breakthrough - "It sounds a bit like science fiction - the drug simply cuts the damaged DNA" • Her father told about the moment he discovered the disease: "As a doctor, I got to tell the news about children with diseases, but as a father - the ground falls away from under your feet" • To…
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