“This is the first time we may have a treatment for the underlying cause in the most common form of cystic fibrosis.”
A locally waged battle against cystic fibrosis has yielded an experimental drug combination that is giving new hope to families affected by a disease that historically has meant death at an early age.
In a clinical trial, Vertex Pharmaceuticals in Cambridge found that a combination of its drug Kalydeco, which won federal regulatory approval only four months ago, and the experimental drug VX-809 substantially improved breathing for some cystic fibrosis patients.
“I never dreamed we’d have this day,” said Joe O’Donnell, who has raised about $250 million for the Cystic Fibrosis Foundation over the past 35 years and lost his 12-year-old son, Joey, to the disease in 1986. “This is tentatively a game-changer. We’re not there yet. But we’re damn close.”
via Boston Herald - Marie Szaniszlo
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