FRAXA Clinical Trial Grant supports scientists studying people with Fragile X syndrome, prioritizing trials of disease-modifying treatments to advance practical therapies and ultimately a cure.
Path to a Cure Collaborative Research Grant supports research teams developing CFTR gene repair, replacement, and delivery technologies to restore CFTR function and advance new cystic fibrosis therapies.
The General Research Grant supports Friedreich’s ataxia investigators in basic research, drug development, and clinical research to advance shared resources, treatments, and a cure.
Supports basic science research into the mechanisms, etiology, and pathogenesis of cystic fibrosis to inform prevention and therapy development.
The SMA Foundation Research Funding program supports projects that accelerate late-stage translational research or clinical trials for new SMA therapeutics.