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5037 results for "muscle disease"
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.
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.
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.
A groundbreaking RNA therapy just succeeded in early trials for a rare muscular dystrophy affecting thousands, and it could become the first treatment that actually modifies the disease's progression. Novartis's del-brax showed clear signs of reducing muscle damage in patients with facioscapulohumeral muscular dystrophy.
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 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.
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 college student diagnosed with a muscle-wasting disease at 13 is now leading research to cure it, winning $50,000 to develop her breakthrough therapy. Her work could help one million people worldwide living with the same condition.
Researchers discovered how to disguise healthy mitochondria so they can slip into diseased cells undetected, extending the lives of mice with a fatal genetic disorder. The breakthrough could one day help children born with rare mitochondrial diseases.
A new treatment for a rare kidney disease just passed a major test, bringing hope to 330,000 patients in the U.S. and Europe. Vertex's drug successfully reduced a key disease marker by half in late-stage trials.
A 21-year-old bioengineering student diagnosed with a rare muscle disease at 13 is now engineering a potential cure for herself and nearly 1 million others worldwide. Héloïse Hoffmann just won a $100,000 fellowship to advance her groundbreaking gene therapy research.
Pearl Jam's Eddie Vedder and Canadian director Matt Finlin created a documentary following the race to cure epidermolysis bullosa, a devastating skin disease affecting children worldwide. The Netflix film captures breakthrough research that could unlock treatments for thousands of rare diseases.
After more than a year of tireless advocacy, a father's campaign has paid off. His 12-year-old son with Duchenne Muscular Dystrophy will finally access a treatment that could help him walk through his entire school career.
For the first time, a gene therapy has shown dramatic results in slowing Huntington's disease, offering hope to 8,000 UK families who've had no treatment options until now. Patients receiving the higher dose experienced 75% less disease progression over three years.
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.
Four leading European research institutes have joined forces to speed up treatments for Alzheimer's, Parkinson's, and other brain diseases affecting millions. The CURE-ND Alliance brings together over 2,500 scientists working to transform how we understand and treat neurodegenerative diseases.
A Nashville family transformed their annual Kentucky Derby party into a powerful fundraiser after their daughter was diagnosed with an incurable nerve disease. Last year alone, they raised $75,000 for research into Charcot-Marie-Tooth disease.
New fitness guidance shows women over 50 can reverse age-related muscle loss with just three weekly strength sessions. The approach helps prevent disease while maintaining independence for years to come.
A groundbreaking cell therapy has successfully treated stiff person syndrome, a rare neurological disorder that causes severe muscle stiffness and mobility problems. If approved by the FDA this summer, it would become the first treatment for the condition and the first personalized CAR-T therapy for any autoimmune disease.
A University of Kentucky scientist is turning Lyme disease bacteria against itself in a promising new treatment approach. The breakthrough comes as tick-borne illnesses have jumped 128% nationwide since 2020.
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