Each year, Patient Worthy covers the American College of Medical Genetics conference, the leading source of both genetic education for health care professionals and a leading source of new information on genetics for all. The only time we witnessed a standing ovation at this very reserved event was at the presentation of the research results for the ultra rare disease Barth Syndrome and the medication elamipretide. At long last the FDA has grated approval for this medication, whose trade name will be Forzinity. We rejoice along with all the Barth Syndrome families, and all the children yet to be born who will benefit from this drug.

We applaud Stealth BioTherapeutics, which persevered through the long approval process, and which has committed to continuing support for the most vulnerable children. In a press release by the Barth Syndrome Foundation, we read:

“This regulatory journey has been a daily exercise of resilience,” says Shelley Bowen, BSF Director of Family Services and Advocacy. “For the past two years our community has tearfully pleaded to be heard in hundreds of Congressional meetings. Today, some of those pleas have been heard. While access for our most vulnerable population remains incomplete, we are deeply grateful for Stealth’s commitment to provide an Expanded Access Program (EAP) for this population to make sure that emergency cases and individuals who are already on the therapy will have access. They are doing right by our community while we continue to push for approval for all ages.”

For more information on Barth Syndrome reach out to The Barth Syndrome Foundation. For more articles regarding Barth Syndrome on Patient Worthy read here.