Health & Fitness
Bay Area Family Shares Frustrations After FDA Rejects Drug For Ultra-Rare Barth Syndrome
"He has severe heart failure, and at the hospital, he was on intravenous medications to help his heart pump," Mom Megan Branagh said.
June 30, 2025
With the FDA recently rejecting a drug application that aims to help those living with Barth Syndrome, one Bay Area family is frustrated by the delay of approved treatments in the rare disease community.
Find out what's happening in San Franciscofor free with the latest updates from Patch.
Approximately 150 people live with Barth Syndrome nationwide, an ultra-rare genetic disorder that affects only males and impacts muscle and heart muscle health.
"He has a severe heart failure, and was admitted to the hospital where he was on intravenous medications to help his heart pump," Megan Branagh, whose son Henry has Barth Syndrome, told CBS News Bay Area.
Find out what's happening in San Franciscofor free with the latest updates from Patch.
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