Curative Therapies Grant supports scientists worldwide researching curative and disease-modifying therapies for Fragile X syndrome, with the goal of bringing practical treatments into medical practice.
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.
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.
PTAC Research Grant supports investigators studying CFTR restoration and gene repair or replacement to inform new therapies for people with cystic fibrosis not served by approved modulators.
General Research Funding from the CMT Research Foundation supports researchers and clinicians pursuing translational and discovery research to advance treatments for all types of Charcot-Marie-Tooth disease.
The SMA Foundation Research Funding program supports projects that accelerate late-stage translational research or clinical trials for new SMA therapeutics.