Natural History for our tiny warriors with MEF2C

Say Hello to our Mefties!

Dear MEF2C Community,

First off, thank you for all your ongoing support in our journey to develop treatments for MEF2C Haploinsufficiency Syndrome (MCHS) (more about the condition here www.mef2cfoundation.com)

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 $1.3 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!

Donate, advocate, spread the word!! As we make progress on our fundraising goal, we will certainly keep this campaign updated and share it with all of you amazing supporters and advocates.

Thank you for being with us on this journey and thank you for spreading the word!

Any funds donated here will reach the MEF2C Foundation in full (no transaction or admin fees involved if donating directly through GoFundMe!) so it is the most efficient way of helping us. Thank you again for all you do to help our tiny warriors!

Lorena - MEF2C Foundation