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Meet Frank Sasinowski, M.S., M.P.H., J.D., our keynote speaker at the 2024 Barth Syndrome International Conference. With vast experience in FDA drug approvals, including for rare diseases, Frank will share insights on clinical trials and future directions. Don't miss his session

Exciting update! U.S. FDA has now determined that the New Drug Application for elamipretide to treat #BarthSyndrome will receive a Priority Review designation. Thank you FDA for recognizing the serious and unmet medical needs of our #BarthSyndrome community. Read more:

Join us for Barth Syndrome Foundation’s virtual townhall on May 23rd at 12pm ET to learn more about the valuable role that the worldwide Barth syndrome community will play in the FDA’s review of Stealth’s new drug application (NDA) for elamipretide. Register here:

Emily Milligan of Barth Syndrome on challenges with developing therapy: "We're being told: 'Go find more people'. They definitionally do not exist. And so as we think about ultra rare drug development, it's feasibility, it's reasonableness, it's fairness. It needs to be

On Sept 6, the U.S. FDA announced AdComm meeting on Oct 10 to review StealthBT NDA for elamipretide. The BSF community can submit letters to share their experiences, open to all globally. Deadline: Sept 26. Learn more: barthsyndrome.org/advocacy/fdaad…

We are excited to share the first video in our #BarthPROGRESS series, highlighting the power of our community. As we await the FDA’s decision on elamipretide (Jan 29, 2025), we’re ready to advocate for broad access if approved. Together, we’ll keep pushing for #BarthPROGRESS

As we enter 2025, we reflect on progress & look ahead to transformative possibilities, like the FDA's Jan. 29 decision on elamipretide—a hopeful milestone for Barth syndrome & beyond. #BarthPROGRESS #Barthsyndrome

In just weeks, the FDA will decide on elamipretide for Barth syndrome (Jan 29, 2025). This could be life-changing for those with Barth syndrome & ultra-rare diseases. Watch the latest #BarthPROGRESS video

The FDA has extended its decision date to April 29, 2025, despite a positive AdComm recommendation. Emily Milligan, BSF Executive Director: "This delay is disappointing, but we’re optimistic elamipretide will be approved this spring." More: bit.ly/42l0M1r

Thank you for being a Champion for those with Barth syndrome!

ketv.com/article/omaha-… #GiftOfProgress #ApproveElamipretide #HopeForBarth #RareDisease #FDAActNow U.S. FDA

wlbt.com/2025/06/05/ran… #GiftOfProgress #ApproveElamipretide #HopeForBarth #RareDisease #FDAActNow U.S. FDA

tmj4.com/news/washingto… #GiftOfProgress #ApproveElamipretide #HopeForBarth #RareDisease #FDAActNow U.S. FDA

atlantanewsfirst.com/2025/06/05/gai… U.S. FDA #GiftOfProgress #ApproveElamipretide #HopeForBarth #RareDisease #fdaactnow

9news.com/article/news/h… #GiftOfProgress #ApproveElamipretide #HopeForBarth #RareDisease #fdaactnow

wsbtv.com/video/local-vi… U.S. FDA #GiftOfProgress #ApproveElamipretide #HopeForBarth #RareDisease #fdaactnow

The Filchak's joined WDUN's "The Martha Zoller Show" on Monday to speak on Hope's Journey and the FDA. Listen and share: bit.ly/4n6NJbG #GiftOfProgress #ApproveElamipretide #HopeForBarth #RareDisease #fdaactnow

podbean.com/media/share/pb… Thank you to the Peña and Wilson families for joining the Kim Wade Show to share their stories and advocate for elamipretide. The interview begins around the 39:40 mark! #GiftOfProgress #ApproveElamipretide #HopeForBarth #RareDisease #fdaactnow

“There’s no question this drug works.” Thank you to the Filchak family for continuing to share your powerful story. Watch and share: bit.ly/40qlTNZ #GiftOfProgress #ApproveElamipretide #HopeForBarth #RareDisease #fdaactnow

The Branagh family continues to advocate for access to elamipretide. Watch and share their story, featured on CBS News Bay Area, to see why this decision matters so much to families living with Barth syndrome. Link to story: bit.ly/40xV4Ya