Welcome!
Meet Gilbert
Hi everyone,
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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”).
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Over the course of the summer we advocated for elamipretide to get FDA approval and finally on September 19, elamipretide was approved!
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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!
