The Barth Syndrome Foundation is thrilled to announce it has been awarded a grant from Cycle 3 of the Chan Zuckerberg Initiative’s (CZI) Rare As One Project. The CZI Rare As One Project supports and unites patient-led organizations working to advance the rare disease field by strengthening communities, building capacity, and promoting collaboration. 

The 10-6 vote to recommend approval signals the advisory committee panel's assessment on elamipretide’s safety and effectiveness with its proposed intended use in Barth syndrome. The committee offers valuable perspective and non-binding recommendations for the FDA to factor in alongside other considerations during approval decisions. The FDA is expected to decide whether to approve the new drug application in January.