Rare Bird Welcomes New FDA Guidance on Rare Disease Treatment

The Rare Bird Foundation was thrilled to be invited to participate in the FDA's announcement of their new framework for accelerating rare disease therapeutics. The team at Rare Bird has been engaging with the FDA early and often, and we are looking forward to continuing to do so as we advance our therapeutic development agenda for MCHS. The FDA's exciting announcement will allow the MCHS community to leverage our RNA-based therapeutic candidate (in development at the Medical University of South Carolina) and our community's rigorous natural data (from the Volare Study at Cornell) to more quickly bring needed relief to our patients.

Read more about the FDA's announcement here:
FDA: FDA Launches Framework for Accelerating Development of Individualized Therapies for Ultra-Rare Diseases | FDA

NPR: https://www.npr.org/2026/02/23/nx-s1-5720948/fda-rare-disease-gene-therapy