Voice of the Patient

Externally-led PFDD Meeting a HUGE Success Because of YOU!

By now you’ve heard…Our externally-led PFDD meeting on July 18, 2018 was a monumental day for our BSF family! Together, nearly 400 people from more than 11 countries united and shared courageous stories from so many incredible members of our community.

We are proud that Barth Syndrome Foundation was the 14th organization of more than 7,000 rare diseases to host a meeting of this kind. We are so thankful to the FDA for selecting Barth syndrome as a condition that requires their attention and focus.

But most of all, we are thankful for you! Many of the clinicians and researchers who joined the meeting said out loud that the BSF community is a tremendous example of patients and families working together for a bigger cause!

What happens next?

The July 18, 2018, PFDD meeting was the first step toward integrating the real-life concerns, struggles, and hopes of people like you, affected individuals and caregivers, into the drug development and FDA approval process. We all want nothing more than to find a way to help people live with fewer struggles and have the chance to live a longer, more fulfilling life.

BSF's Voice of the Patient report of the PFDD meeting is due out in late December/early January and will serve as a critical summary on the experience of living with Barth syndrome and clinical outcomes that are meaningful to our community. Be sure to subscribe to BSF E-News to receive your copy.

The Voice of the Patient report is critical for researchers to submit new therapies to the FDA for regulatory approval. It will also inform the clinical trial endpoints that are important to our community. For more information on the Voice of the Patient report, visit the FDA's website.

Powered by Firespring