SAN FRANCISCO (KPIX) — 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.
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…