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6 results for "sarepta"
After a difficult year, biotech company Sarepta Therapeutics is bouncing back with early clinical trial results showing two new muscular dystrophy treatments appear safe and effective. The promising data offers hope for patients with rare muscle-wasting diseases who desperately need new treatment options.
Scientists just achieved a major breakthrough in treating two rare muscle diseases that currently have no cure. Early trials show a new delivery method gets medicine directly into muscle cells without serious side effects.
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 safer gene therapy for Duchenne muscular dystrophy just hit a major milestone, bringing hope to families facing this fatal muscle disease. The treatment could offer a better option than current therapies linked to serious side effects.
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 groundbreaking treatment for Huntington's disease just cleared a major hurdle, bringing hope to 240,000 Americans affected by this devastating condition. The experimental therapy could become the first disease-modifying treatment for a disorder that has had none.