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7 results for "fshd"
A groundbreaking treatment that switches off a faulty gene is showing early success in 12 people with a rare form of muscular dystrophy. Instead of losing muscle as expected, patients are gaining strength.
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.
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.
Scientists in Japan have discovered that simple iron supplements can significantly improve muscle strength in mice with a rare form of muscular dystrophy, offering hope for thousands of patients with no current treatment options. The breakthrough works by correcting iron imbalances in muscle tissue, not by fixing the underlying genetic cause.
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 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 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.