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4 results for "muscle disease"
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.
A nonprofit and patient foundation just committed $7.65 million to develop a gene therapy for a rare muscular dystrophy with no current treatments. The partnership will take the therapy from initial design all the way to human clinical trials.
A groundbreaking treatment for myotonic dystrophy type 1 reduced toxic proteins by 40% and improved muscle function in patients who've never had disease-modifying options. The New England Journal of Medicine just published results that could lead to the first approved therapy for this progressive, often fatal disease.
Patients with a debilitating muscle disease saw symptoms nearly disappear after just six weeks of treatment—and the relief lasted a full year. The breakthrough could transform care for multiple autoimmune conditions.