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I’m pleased to share encouraging news. The FDA has granted accelerated approval for Forzinity, the first treatment ever for Barth syndrome, a rare, life-threatening disease that primarily affects boys.

Barth syndrome can cause severe heart failure, muscle weakness, and tragically shortened life expectancy. For years, families have pleaded for options.

Earlier this year, I led a letter with my SC colleagues urging FDA action. Thanks to the persistence of patients, families, advocates, and physicians, this hope is now a reality.

https://www.fda.gov/news-events/press-announcements/fda-grants-accelerated-approval-first-treatment-barth-syndrome?fbclid=IwY2xjawNQw_1leHRuA2FlbQIxMABicmlkETE4Zk5qVThFbjRsbk15RDJvAR6sKrxGYBNQtAYLSQHO1WQ0QG_YOdVUpZSOBBjzJ0w1Ufe36Dh9SwJOD0Et_w_aem_rv-P3h3Br1XMeqxaUfD6Ag