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6 results for "duchenne muscular dystrophy"
Boys living with Duchenne muscular dystrophy in England now have access to a new treatment that could extend their ability to walk by up to five years. The NHS has approved givinostat, offering fresh hope to families facing this devastating muscle-wasting disease.
Pharmaceutical company Roche announced a new clinical trial that could bring a life-changing gene therapy for Duchenne muscular dystrophy to young patients across Europe. The move comes after extensive feedback from families and regulators who want to see this treatment option become available.
A mother who raised $1.3 million to fund a controversial treatment for her son's fatal muscle disease just saw clinical trial results that stunned experts. The experimental drug uses a counterintuitive approach: fixing broken genes by breaking them a little more.
A Staten Island father is leading 15 runners through 80 miles of Arizona desert to fund a cure for the disease affecting his 16-year-old son. JAR of Hope has taken teams around the world to raise money for Duchenne muscular dystrophy research.
Boys with Duchenne muscular dystrophy are showing sustained improvements three years after receiving Elevidys gene therapy, demonstrating the treatment can slow progression of this muscle-wasting disease. The results offer hope to families facing a condition that typically causes rapid physical decline around age 9.
A gene therapy called ELEVIDYS is helping young boys with Duchenne muscular dystrophy maintain their ability to walk, run, and stand years after a single treatment. Three years after receiving the therapy, treated children showed 70% less decline in muscle function compared to untreated children.