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7 results for "muscle disease"
A Yale scientist is discovering why some patients with myasthenia gravis don't improve with standard treatments, opening doors to personalized therapies. Her groundbreaking work could help doctors match the right medicine to each patient's unique disease pattern.
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 new high dose version of an SMA treatment just won FDA approval, offering faster results for babies and children fighting this devastating muscle disease. The breakthrough gives families a more powerful option against a condition that once had no treatment at all.
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.