Find uplifting stories about heroes, innovations, and solutions
7 results for "rare disease cure"
Babies born without vital gut nerves may soon receive a permanent cure instead of repeated surgeries. UK researchers created lab-grown stem cells that restore digestive function in just six days.
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.
Jeff Allen won the largest cash prize in game show history and is using it to fund research for his son's rare brain disease. His efforts could unlock treatments for millions with Alzheimer's and Parkinson's.
A 19-year-old from British Columbia became the first person ever cured using "prime editing," a breakthrough gene therapy that fixed a fatal immune disorder. The treatment freed him from daily medication and the constant fear of life-threatening infections.
When federal funding for rare diseases falls short, grieving parents are stepping up to fund research themselves and saving lives. Their donations are now achieving medical breakthroughs doctors once thought impossible.
After a decade of stagnation where only 5% of rare diseases had treatments, Europe is overhauling outdated regulations to bring hope to 30 million patients. New pathways are finally treating ultra-rare conditions differently than common illnesses.
Jeffrey Randall Allen won $10 million on Amazon's Beast Games and immediately directed millions toward researching a cure for his son Lucas's rare genetic disorder. His mission is bringing unprecedented funding and attention to Creatine Transporter Deficiency, a condition that affects how the brain accesses a critical nutrient.