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17 results for "rare disease research"
After nearly dying five times from a mystery illness, Dr. David Fajgenbaum turned to artificial intelligence to save his own life—and now he's finding treatments for thousands of other forgotten patients. His AI system has already identified potential cures for over 60 rare diseases that drug companies won't touch.
Researchers at the University of Michigan have discovered what causes a rare condition that makes walking so painful patients crawl instead. An FDA-approved cream has already shown promise in early trials.
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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.
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.
Children with a devastating form of epilepsy may soon have new hope thanks to promising trial results from an experimental treatment. Praxis Precision Medicines announced their drug elsunersen successfully reduced seizures in early testing for SCN2A-related epilepsy.
When someone told a young woman she was "too close" to her family's disease to become a researcher, Jill Briceño had the perfect response. Her answer, inspired by mastering Cuban cooking, could change how we think about rare disease research.
Researchers discovered how to disguise healthy mitochondria so they can slip into diseased cells undetected, extending the lives of mice with a fatal genetic disorder. The breakthrough could one day help children born with rare mitochondrial diseases.
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.
Jeff Allen won $10 million on "Beast Games" and is using his prize to fund research for his son Lucas's rare genetic disorder. His efforts could unlock treatments for millions with Alzheimer's and Parkinson's.
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.
Families fighting Huntington's disease celebrated a breakthrough this Valentine's Day as researchers announced the first treatment for the deadly brain condition. The discovery brings renewed hope to a community that has waited decades for medical progress.
The UK and Japan just announced a major science collaboration worth over £27 million that will advance gene therapy, quantum computing, and next-generation communications. The partnership promises breakthrough treatments for rare diseases and cutting-edge technology that could transform healthcare and digital connectivity in both nations.
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 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%.
Scientists at the University of Twente and Radboudumc are building a working human retina on a microchip, eliminating the need for animal testing while speeding up treatment for rare eye diseases. They've already successfully combined the first two retinal layers, something never achieved before.
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.