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Scientists just achieved a major breakthrough in treating two rare muscle diseases that currently have no cure. Early trials show a new delivery method gets medicine directly into muscle cells without serious side effects.
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.
After their daughter was diagnosed with a rare disease stealing her sight and hearing, one family turned heartbreak into action by launching a foundation to fund personalized gene therapy research. Their work could help thousands of families facing similar battles.
After years without hope, Susannah Rosen received a breakthrough treatment for a rare neurodegenerative disorder that was slowly killing her brain cells. A nonprofit foundation now provides the custom-made medicine free for life to patients with ultra-rare diseases.