Funding the science that could change everything.
There's no approved cure for L-CMD yet. Researchers are making real progress in the lab, and we want to help that progress reach children like Archie.
Where research is heading
Here's a quick, plain-language look at some of the approaches scientists are exploring. Most of this work is still in early, preclinical stages.
Gene editing
Scientists are testing CRISPR-based tools in lab models to switch off or correct the faulty copy of LMNA. Early mouse studies have shown longer survival and better heart function.
PreclinicalBase editing
A precise "spell-check" for DNA. In 2025, researchers used base editors to correct LMNA variants in mice, including one linked to CMD, and improved heart function and lifespan.
PreclinicalNew delivery methods
Getting therapies into muscle and heart cells is a major hurdle. Non-viral delivery systems are being developed to carry gene-editing tools where they need to go.
PreclinicalNatural history and registries
Understanding how L-CMD progresses over time is essential for designing clinical trials. Family participation in studies and registries makes that possible.
OngoingLearn more: Base editing in LMNA mouse models (PNAS, 2025) · Cure CMD research · L-CMD Research Foundation · ClinicalTrials.gov
How we'll invest in research
Fund what's promising
We'll direct funds to scientific work with a real path toward treatments for L-CMD.
Work together
We plan to partner with established researchers and rare disease organizations instead of duplicating their work.
Report openly
We'll share where donations go and what they help accomplish.
Are you an L-CMD researcher?
We'd love to learn about your work and explore how we can help.