Rare Community Profiles: For Rare Disease Week, Barth Syndrome Advocates Took to Capitol Hill to Urge the FDA to Review NDA for Elamipretide

• Post author:Jessica Lynn
• Post published:March 26, 2024
• Post category:Barth Syndrome

  Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…

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FDA’s Refusal to Review Rare Disease Treatment Denies Patients’ Right to Try

• Post author:Patient Worthy Contributor
• Post published:March 19, 2024
• Post category:Barth Syndrome

This opinion piece was provided by Kevin Woodward of Phoenix, Maryland. Kevin is a member of the Barth Syndrome Foundation board of directors. The role of the U.S. Food and…

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World Orphan Drug Congress USA 2023: The Challenge of Ultra-Rare Disease Drug Development

• Post author:James Moore
• Post published:June 15, 2023
• Post category:Barth Syndrome/Timely

The World Orphan Drug Congress USA 2023 was held from May 23-25, 2023 at the Gaylord National Resort & Convention Center in National Harbor, MD. This conference is focused solely…

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EMA Grants Orphan Drug Designation to Elamipretide for Barth Syndrome

• Post author:Kendall Mason
• Post published:June 29, 2022
• Post category:Barth Syndrome

According to a recent news release from PR Newswire, the European Medicines Agency (EMA) has recently awarded elamipretide its Orphan Drug designation for the treatment of Barth syndrome. This treatment,…

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Barth Syndrome Chairman Appeals to the FDA For Appropriate Guidelines When Approving Drugs for Ultra-Rare Diseases

• Post author:Rose Duesterwald
• Post published:September 24, 2021
• Post category:Barth Syndrome

According to an article submitted to StatNews by members of the Barth Syndrome Foundation, the FDA approved the drug Aduhelm in accordance with its accelerated approval program. Although the treatment…

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New Fellowship Will Advance Barth Syndrome Research

• Post author:Kendall Mason
• Post published:June 4, 2021
• Post category:Barth Syndrome

Rare disease research is often lacking due to a lack of funding, interest, or awareness. To combat this, the Barth Syndrome Foundation (BSF) and American Heart Association (AHA) have joined…

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Alexander’s Story: Diagnosed with Barth Syndrome After Losing Brother

• Post author:Kendall Mason
• Post published:May 4, 2021
• Post category:Barth Syndrome

One-year-old Alexander was diagnosed with Barth syndrome before he was born; his parents knew to test for the condition after their son Elias passed away from complications in 2018. While…

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University of Florida Study: Barth Syndrome Gene Therapy Preclinical Results Look Good

• Post author:Jean Martell
• Post published:December 18, 2018
• Post category:Barth Syndrome

A University of Florida research team has announced its results of a gene replacement therapy in the treatment of Barth syndrome, which delivered promising results in its initial phase of testing.…

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