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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

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