Recently, Lynda Sedefian, parent of two sons with Barth syndrome, Erik and Derek, met with Rep. Paul Tonko [D-NY-20] to discuss his support of the Barth Syndrome Awareness Day Resolution in the US House of Representatives (H.Res.276). Lynda shared her experience as a mother of two individuals with Barth syndrome, the impacts of the genetic disease, and...
Disability is often viewed as a condition observable at a distance without any interaction, like someone using a wheelchair, a cane, or other assistive devices. Out of the 26 million Americans who have a severe disability, only 26% use an assistive device. The remaining majority have very little or no visual indications of disability, requiring intentional disclosure to bring awareness of their disabilities.
Conversations around disability are necessary to not only advance access and inclusion for people with disabilities, like Barth syndrome, but empower...
The Barth Syndrome Foundation (BSF), including BSF representatives as well as key clinical disease leaders, held an important workshop with Dr. Norman Stockbridge (Director of the Division of Cardiology and Nephrology in CDER at the FDA) and over 30 other representatives of different FDA centers, offices, and divisions on July 29, 2022. The stated purpose of this workshop was...
We at BSF express our heartfelt gratitude to Bryan D, Steven G and Walker B for recently meeting with legislators in their home States. All three volunteer ambassadors participated in a national campaign for better U.S. Food and Drug Administration (FDA) review processes for ultra-rare indications, like Barth syndrome.
Efforts by Bryan, Steve, and Walker were focused on greater transparency and...
Boston, June 15, 2022 - Stealth BioTherapeutics (Stealth) announced yesterday that the U.S. Food and Drug Administration (FDA) has granted the company a meeting to discuss a possible new drug application (NDA) for elamipretide as a potential treatment for Barth syndrome. The company intends to present new data collected during the Open Label Extension (OLE) period.
Barth syndrome, unfortunately, is one of the more than 6,500 rare diseases without an approved therapy. As the only organization globally representing the Barth syndrome community, BSF has been on the front lines with the FDA, advocating for effective, fair...
BSF's Executive Director, Emily Milligan, wrote to Stealth BioTherapeuitic's leadership after the FDA refused to file the NDA for elamipretide. To read the full letter, click the title above.
BSF's Board Chair, Kate McCurdy, recently wrote to leadership at the US Food and Drug Administration (FDA) regarding the FDA's refusal to file Stealth BioTherapeutics' NDA for elamipretide. Click the title above to read the full letter.
We were honored to participate in the EveryLife Foundation’s prestigious 13th Annual Rare Disease Scientific Workshop on October 21, 2021-- the same day that we learned that the FDA had refused to review Stealth BioTherapeutics’ New Drug Application (NDA) for elamipretide in Barth syndrome. BSF’s Executive Director, Emily Milligan, gave a highly relevant and impactful 10-minute talk regarding suggestions she has resulting from our community’s experience with the regulatory process for elamipretide.
The Barth Syndrome Foundation (BSF) is deeply disappointed by the US Food and Drug Administration’s (FDA) refusal to file Stealth BioTherapeutics’ (Stealth) submission of a New Drug Application (NDA) for elamipretide as a treatment for those living with Barth syndrome.
While the decision is a setback for the Barth syndrome community, we recognize...
Members of the Barth syndrome community:
August 2021 saw the achievement of a shared milestone – the first submission of a new drug application for elamipretide by our team at Stealth, representing the first new drug application for any investigational product for Barth syndrome. This was a Herculean effort...
Inconsistencies in the FDA’s approach to operationalizing the 21st Century Cures Act stand to treat individuals with Barth syndrome qualitatively differently from those with other diseases, such as Alzheimer’s, and points to a critical need for consistent application of patient perspectives in the review process across agency divisions and regulators.
More than 600 advocates from 250 patient organizations came together virtually for Rare Disease Week on Capitol Hill that took place July 14-22, 2021. Shelley Bowen, BSF’s Director of Family Services and Advocacy, was among those voices advocating for the Barth syndrome and other rare disease communities. "It's important to step up, show up and speak up with peer advocacy groups...”
Today is an important milestone in BSF’s advocacy journey. Ten individuals from the Barth syndrome community are speaking directly to FDA in a closed listening session to share perspectives on access to effective treatments for Barth syndrome. While today’s session is not open to the public, we intend to share insights gained from this important meeting later this month...
TREND analyzed the BSF Facebook group and listserv for the report to construct this report detailing patient and caregiver perspectives.
Industry stakeholders can use this data to: establish disease natural history, identify unmet therapeutic needs, understand quality of life issues, design better clinical trials, and build cases for patient-centered regulatory approvals.
Community members will find the data useful to advocate, inform medical and support teams, educate family members, catalyze research, and spread awareness.
Click the image to access the full report.
BSF is very proud to share the Voice of the Patient report from the July 2018 externally-led Patient Focused Drug Development meeting. This comprehensive report truly reflects our community’s experience of being affected by Barth syndrome. We encourage you to read this and share widely! #powerupbsf #poweruppfdd
