This summer you published my appeal for help to contact the FDA about a lifesaving drug for Barth syndrome (“Call for FDA drug approval,” 6/12/25).
After 10 years postponing approval of this drug, the FDA finally got it right!
My grandson has Barth syndrome. He is a trial patient and will be a part of the trial for his lifetime.
His mother joined our small Washington, D.C., protest on Aug. 20. She met with U.S. Food and Drug Administration Commissioner (FDA) Dr. Martin Makary and his staff with three other mothers by invitation on Aug 22. Unbelievable. She is Rrose LaMay …
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This summer you published my appeal for help to contact the FDA about a lifesaving drug for Barth syndrome (“Call for FDA drug approval,” 6/12/25).
After 10 years postponing approval of this drug, the FDA finally got it right!
My grandson has Barth syndrome. He is a trial patient and will be a part of the trial for his lifetime.
His mother joined our small Washington, D.C., protest on Aug. 20. She met with U.S. Food and Drug Administration Commissioner (FDA) Dr. Martin Makary and his staff with three other mothers by invitation on Aug 22. Unbelievable. She is Rrose LaMay and lives in Conshohocken with her adorable son, Giancarlo.
The drug was finally approved last Friday. This is a testimony to peaceful advocacy and persistence. It is a testament to science, families’ stories, and the FDA. These children with Barth syndrome will have a chance at a better life with this drug. Further research will expose more uses.
Using our voices is more important than ever.
Thanks from a very relieved grandmother.
Margaret Bradley
Chestnut Hill
