A letter from our founder:

Dear Supporter,

I am excited to share with you our first annual report, capturing our incredible progress over the last year. Thanks to your support, we have been able to achieve so much—not just in developing the first two therapeutic candidates for MEF2C Haploinsufficiency Syndrome but also launching the first-ever longitudinal natural history/clinical readiness study, our toxicology/safety work plans, and our community's first engagement with the FDA. I hope you feel some pride in reading about our community's accomplishments. Your involvement and support have made this all happen.

Over the past year, with the help of our partners and supporters like you, the Rare Bird Foundation has moved mountains for children with MCHS. We’ve shown that our therapeutics can increase the level of MEF2C expression in mouse and human cells, and our clinical readiness study—the Volare Study—has already begun its second round of patient visits. We’ve also organized our community's first formal engagement with the FDA, continued characterizing three distinct MCHS mouse models to understand the nuances of various MEF2C mutations, and in the next couple months we will deliver our community's first iPSC lines and toxicology tests to optimize our treatments.

Your support has enabled this ground-breaking work, and we couldn't have done it without you. For the first time ever, families whose children have received an MCHS diagnosis have hope that a treatment is on the horizon.

Of course, we have much still to do. In keeping with our #strive4five mission, we are committed to seeing our therapeutic candidates through the next 12 months of critical work--ensuring their efficacy, safety, deliverability, and regulatory approval--so we can get from lab to clinic and ultimately to patients worldwide within five years (that's three years from now!).

We have accomplished so much with your help, and we are excited to be embarking on a range of new partnerships and opportunities—from industry and academic partnerships to get us the manufacturing materials we need quickly and at no cost, to cooperative research agreements to get all the necessary expertise in drug development, to additional clinical readiness sites so our global population can take part, to new translational projects to address additional avenues of relief for our children.

Our efforts over the next 18 months will be critical to our mission. Your support powers that work. Please donate to our Wings of Change campaign to help us raise $100K before the end of the year—$50,000 by Giving Tuesday and the remainder before December 31st. Two matching donors will help us reach this goal! Read more at the buttons on the right, and spread the word.

We can’t thank you enough.

With gratitude,

Dr. Isra J. Bhatty
MCHS Parent and Founder, Rare Bird Foundation