Welcome!
Project for Awesome 2026
Voting is open until Feb 18 @ 12pm ET!
Ready to change lives?
Vote for Heart of PPA2 ❤️ (@heartofppa2) during Project for Awesome 2026! #p4a2026
Project for Awesome (@projectforawesome) is an event each year where people come together to nominate non-profits that they think deserves to win grant funding! We made a video to nominate Heart of PPA2 and it is up on the P4A website now!
This year during the 48 hour livestream from Feb 13-15, people from around the world donated to raise over $4 million dollars! Donations and voting will be open until February 18 at 12pm ET. Once voting and donating closes, 50% of the total funds raised will be split between Partners in Health and Save the Children. The other 50% will be evenly divided between the top voted non-profits!
If we all work together and spread the word, Heart of PPA2 could be one of the non-profits to win grant funding! You only get ONE vote, so cast your vote for Heart of PPA2 and then tell all your friends to vote too!
YOU can be a part of the chance to fund research that will lead to treatment and a cure! Your support during Project for Awesome 2026 will help this incredible group of families win grant funding for research to develop treatment for their children and for future children diagnosed with PPA2 deficiency.
Go to Projectforawesome.com and search for “Heart of PPA2” and cast your vote!
Meet Gilbert
Hi everyone,
This is our baby boy Gilbert. Shortly after he was born, Gilbert was diagnosed with a very rare genetic condition called Barth syndrome. Due to heart failure, we were in the hospital cardiac care unit with Gilbert for 80 days. The main reason he’s home right now is because of a new drug called elamipretide (pronounced “EL-EM-EP-REE-tide”).
Over the course of the summer we advocated for elamipretide to get FDA approval and finally on September 19, elamipretide was approved!
This website was originally designed as a hub for information about how to advocate for Gilbert. During our advocacy, we connected with some of the most incredible rare disease families so now we will be highlighting ways that you can support our friends!
Thank you for joining us!
Our Dazzle
Did you know that a herd of zebras is called a dazzle? Zebras are symbolic of the rare disease community because in medicine people are often told “when you think hoofbeats think horse, not zebra.” In other words, the problem is probably a more common diagnosis. For Gilby and his friends though, it turned out that the diagnoses they received were the rare ones.
While Gilby and his friends have different types of rare diseases, their families share the same determination to do whatever it takes to save their children's lives. Collectively these families have started non-profits, raised money to go toward treatments, met with congressional leaders, advocated for FDA approval, and done countless new stories to try and raise awareness about the urgency of their children's diseases.
To learn more about Gilby's friends and how you can help click on their pictures and follow them on social media!
