Our most significant projects are listed here. This is where you will find information on where we need your help the most, right here, right now. To learn more about ways to fundraise visit our Fundarising and Events page or simply hit the donate button! .
Campaigns
Natural History
Our team of experts at the Medical University of South Carolina is well on its way to executing "Pathways to Hope," which we launched with your amazing help last year (help-develop-treatments-for-mef2c-change-lives). We are thrilled by the success we have seen in the lab so far, and anticipate that we will see success in mouse models in just a few months.
Before we can take any of these promising treatments to our children, however, we need to check a few regulatory boxes. One of the biggest and most time-sensitive of these checkboxes is the Natural History, which creates baseline scientific measures that ensure that the effects of a future treatment can be accurately measured. This is an absolutely critical piece of the process that is required for clinical trial readiness.
To complete the natural history, which typically takes three years, we need to raise $1 million. We hope your support will go a long way in this fundraising, which we also seek to support through additional funding sources. We know this is a heavy lift, but we think our amazing global community can help us achieve this goal!
https://www.gofundme.com/f/pathways-to-hopes-natural-history
https://www.givengain.com/cc/natural-history-for-our-tiny-warriors-with-mef2c/ (great platform if you are donating internationally)
“Pathways to Hope”: Drug Discovery
This successful world-wide campaign has managed to raise the required funds, in order to drive therapeutics development, for MEF2C Haploinsufficiency. The MEF2C Foundation has been working with Dr Cowan, a global leader in MEF2C research from the medical university of south carolina, to establish (and launch! link here) a research program that could get at least one high-chance-of success treatment ready for clinical trials in the next 3 years. As of today, a treatment for this profound condition does not exist, but this will change thanks to this drug development program! Read all about it here.