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19119 results for "rare disease research"
A Nashville family transformed their annual Kentucky Derby party into a powerful fundraiser after their daughter was diagnosed with an incurable nerve disease. Last year alone, they raised $75,000 for research into Charcot-Marie-Tooth disease.
A new digital platform in Sweden is making it easier for patients to share their health data for rare disease research, with over 5,000 people now registered to help scientists find answers. When patients feel informed and involved, their willingness to contribute soars to 94%.
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A Colorado scientist's breakthrough research is transforming treatment for children with a devastating rare disease, bringing relief after years of pain. Two patients are already showing remarkable improvement from a new therapy approach.
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.
A nonprofit is using AI to match 4,000 existing drugs with 18,000 diseases in less than a day, potentially bringing treatments to millions suffering from rare conditions. Every Cure has already identified 10 promising treatments since 2022.
After losing her sister to Alström syndrome at 28, Rocky Tucker now leads an international organization bringing researchers and families together to find a cure. This May, Fort Worth hosts a global conference for one of the world's rarest diseases.
A mother whose daughter was diagnosed with a rare genetic disorder created an AI platform that saves families 53 hours a week and accelerates treatment research by up to 50%. Citizen Health now helps over 8,000 patients across 350 rare diseases navigate care and connect with others facing the same challenges.
A Tennessee family is turning their son's rare disease diagnosis into a community rally for hope and research funding. The Walk for a Cure event next Saturday will support gene therapy research for PKAN, a terminal neurological condition affecting only one to three people per million worldwide.
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.
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.
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.
A Philadelphia nonprofit founded by a doctor who survived a rare disease just landed $76 million to help millions of patients find treatments hiding in plain sight. Every Cure's AI tool searches thousands of existing drugs to find new uses for rare diseases that lack treatments.
A Colorado scientist's decade of detective work just gave hope to families battling Blau syndrome, a genetic disease affecting fewer than 1,000 Americans. Two patients are already seeing remarkable results.
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.
After losing his oldest son Nick to neurofibromatosis, billionaire Dan Gilbert is pouring $50 million annually into research for the rare disease that affects 170,000 Americans. The Gilbert Family Foundation has turned grief into action, raising $12 million in a single night while creating lasting tributes that keep Nick's memory alive.
Scientists at Stanford, MIT, and Cambridge are using Google DeepMind's Co-Scientist AI to accelerate breakthroughs in liver disease, aging, and infectious diseases. The system helps researchers digest decades of studies in days, spotting connections humans might miss.
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 new drug for cutaneous sarcoidosis exceeded all expectations in a clinical trial, improving patients' severe skin lesions far beyond what researchers hoped for. The breakthrough offers hope to thousands living with a rare disease that has few treatment options.
Over 100 researchers used Google DeepMind's new AI tool to tackle 29 undiagnosed rare diseases, revealing clues hidden in the 98% of DNA that scientists barely understand. This breakthrough could help 350 million people worldwide living without answers.
Nigeria's medical research institute has unveiled a biobank containing over 400,000 biological samples that could revolutionize how the country understands and treats diseases. The massive collection offers locally generated data that researchers say will lead to better health outcomes for millions of Africans.
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