Find uplifting stories about heroes, innovations, and solutions
8 results for "friedreich ataxia"
Australian researchers discovered a compound from broccoli could treat Friedreich ataxia, a rare genetic disease with no cure that affects 260 Australians. The affordable treatment might help patients live longer, healthier lives.
Scientists at Brunel University and UCL have developed a stem cell treatment that could prevent Friedreich's ataxia before symptoms appear. In mice, the therapy restored near-normal protein levels and improved movement, offering hope for children facing this devastating condition.
Scientists discovered that brain signals researchers have relied on for decades may have been misleading them, opening new paths to treat tremors, dystonia, and ataxia. This breakthrough could transform how millions of people with movement disorders receive treatment.
Scientists at Carnegie Mellon University have discovered a breakthrough approach to target the root cause of myotonic dystrophy type 1, the most common form of adult muscular dystrophy. The precision therapy could finally offer hope to patients with a disease that currently has no effective treatment.
Scientists at Carnegie Mellon University have discovered a precise way to target toxic RNA that causes myotonic dystrophy type 1, the most common adult-onset muscular dystrophy affecting 1 in 2,300 people worldwide. The breakthrough could lead to treatments with fewer side effects for this currently incurable disease and other devastating genetic disorders.
Adults with a rare genetic disease called cystinosis no longer need dozens of pills each day after receiving a groundbreaking one-time gene therapy. Five years later, most patients show stable or improved health across multiple organs.
Scientists at Johns Hopkins discovered that a "dormant" brain protein is actually a powerful switch that could lead to new treatments for anxiety, schizophrenia, and movement disorders. The breakthrough reveals how these mysterious proteins actively control how brain cells communicate.
Scientists analyzed nearly 3,000 patients and discovered that a gene previously thought to only affect immunity actually causes a rare neurological condition. The breakthrough could help diagnose people who've lived years without answers about their movement difficulties.