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6649 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.
Scientists are using a safer twist on gene editing to treat diseases by flipping genetic switches instead of cutting DNA. The first trial results for a rare muscular disorder just arrived, and they're opening doors for conditions from high cholesterol to inherited diseases.
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 new medicine called Pasatru just won FDA approval for fibrodysplasia ossificans progressiva, a devastating disease that turns muscle into bone. The breakthrough could help patients maintain mobility and live longer after three decades of research.
Researchers at Emory University discovered that muscle cells repair themselves in a way scientists thought impossible, solving a puzzle that's stumped experts for decades. The breakthrough could lead to better treatments for heart failure and other muscle diseases.
For the first time ever, children and adults with Alexander disease have a treatment that attacks the root cause of their condition, not just the symptoms. The FDA's approval of zilganersen gives hope to families facing a disease that has always been fatal.
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.
Scientists are discovering that exercise does more than improve movement for Parkinson's patients. Muscles release protective molecules called "exerkines" that travel to the brain and may actually slow disease progression.
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.
A doctor who survived a rare disease now uses artificial intelligence to find unexpected treatments that help patients like Kaila Mabus, who was told she might not survive. His AI system tests 75 million possible drug-disease combinations in minutes.
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.
University of Queensland researchers have developed a breakthrough drug that could transform Motor Neurone Disease from a terminal diagnosis into a manageable chronic condition within five years. The discovery unlocks a previously untargetable immune receptor that plays a key role in neurodegenerative diseases.
Scientists at the University of Colorado Boulder are studying pythons' remarkable ability to grow and shrink their hearts without damage, hoping to unlock treatments for human heart disease. The snakes can fast for months while maintaining muscle strength and boost their metabolism up to 40 times after eating.
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.
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