Find uplifting stories about heroes, innovations, and solutions
234 results for "rare disease"
A groundbreaking gene therapy called Otarmeni is restoring hearing to children born deaf, and the company is offering it completely free in the U.S. Kids who couldn't hear a lawnmower next to them can now hear whispers within months of treatment.
A targeted therapy for advanced gastrointestinal cancer shrank tumors by 30% or more in 61% of patients who tried it as their first treatment. The breakthrough could offer hope to those facing drug resistance in a cancer that affects thousands worldwide.
A breakthrough medication for a rare hereditary condition that causes tumors throughout the body is now affordable for hundreds of Australians. Patients who lived surgery to surgery finally have hope for their futures.
After two rare diseases nearly killed her, Fleur Marks wrote a book helping overachievers find balance before burnout breaks them. Her promise to help others is saving lives while she fights for her own.
A second-grader ran a lemonade stand for a week to help fund gene therapy research for his best friend Jack, who has a rare genetic disease. The community rallied around the effort, doubling the $5,000 goal.
The FDA just approved a new treatment for adults with blastic plasmacytoid dendritic cell neoplasm, an extremely rare and aggressive blood cancer. This marks new hope for patients facing a disease with very few treatment options.
A small town in Saskatchewan is rallying behind families affected by cystic fibrosis, with youth bakers and community volunteers kicking off a week of heartfelt fundraising. Their efforts are building connections that organizers say matter just as much as the dollars raised.
Louis was diagnosed with a rare tumor at 18 months old that left him blind and partially deaf, but he refused to let it stop him from pursuing his passion. Today, he's built a successful career in music, proving that disability doesn't define destiny. ##
Years of relentless fundraising by an Australian family living with neurofibromatosis has led to a breakthrough discovery that could transform treatment into something as simple as diet changes or probiotics. Melbourne researchers found crucial gut bacteria differences in NF1 patients, opening doors to affordable therapies for cognitive and behavioral symptoms.
A free artificial intelligence system can now match rare cancer patients with potential treatments in minutes, offering new hope for thousands whose tumors have few dedicated clinical trials. The open-source tool worked for all 260 sarcoma patients tested.
A rare muscle disease that once offered families almost no hope now has multiple new treatments helping kids stay stronger longer. Gene therapies and innovative medications are changing what's possible for children with Duchenne Muscular Dystrophy.
Scientists at UT Austin mapped the protein networks of a billion-year-old organism that gave rise to all complex life, uncovering hundreds of genes connected to human diseases. The breakthrough has already helped identify genetic causes of three rare disorders.
A University of Nevada scientist has discovered a promising drug that could help protect muscles in patients with deadly Duchenne muscular dystrophy. After 20 years of research, Dean Burkin's team is preparing to test their breakthrough therapy in human trials.
Chinese scientists unveiled a supercomputer platform that screens millions of drug compounds a million times faster than previous technology. The breakthrough could revolutionize how we find treatments for cancer, rare diseases, and emerging pandemics.
Years of tireless fundraising by teacher's aide Zoe Petropoulos and her family have helped fuel a breakthrough that could transform treatment for a genetic disorder affecting thousands. The discovery could lead to treatments as simple as probiotics.
A rice-sized implant with living cells is now FDA-approved to slow vision loss in MacTel patients, offering hope to thousands losing their sight. For the first time, people with this rare retinal disease have a real chance to preserve their independence.
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.
When Shannon was given 6-14 months to live with stage 4 brain cancer, her scientist father refused to accept it. Now he's building a database to help other families find hope.
Children born with epidermolysis bullosa, a painful genetic disorder causing skin to blister at the slightest touch, now have real treatment options for the first time. Three new FDA-approved therapies are healing wounds that doctors could only bandage before.
A Victoria boy with a rare disease is throwing a country party to fund the cure that could save his life. Simon Hoskins hopes his second annual Hoedown for Hope will raise $50,000 for MPS research.
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