Accelerating Gene Therapy Research for Rare Diseases
Cure Rare Foundation helps close the funding gaps that keep promising gene therapy research from reaching patients with rare neurological diseases.
Who We Are
Cure Rare Foundation was founded by families directly impacted by rare disease.
We focus on a critical problem in rare-disease research: promising science can stall because funding for the next step simply isn’t there. Cure Rare Foundation helps fill those gaps so researchers can keep moving toward treatments, and ultimately…cures.
Gene Therapy, Explained Simply
Many rare genetic diseases begin with a problem in a gene—the body's instruction manual.
Gene therapy aims to address disease at its genetic source. Depending on the approach, it may replace a faulty gene, repair genetic instructions, or help cells compensate for what isn't working correctly.
For many rare neurological diseases, approaches like these offer the possibility of treating the underlying cause, not simply managing its effects. Cure Rare exists to help promising research keep moving toward that possibility.
Why It Matters
Thousands of rare diseases affect families worldwide, but promising research can struggle to secure the funding needed to move from one critical stage to the next.
Families cannot wait. Science cannot stall. Cure Rare exists to help close that gap…now!
Contact Us
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