FDA to hold Jan. 22 hearing on muscular dystrophy drug
Bay Ridge boy is part of clinical trial
The parents of a Bay Ridge boy suffering from a rare form of muscular dystrophy are bringing their fight to save his life to Washington D.C. next month.
Manni and Dayna Scarso said they will speak at a Food and Drug Administration (FDA) meeting in the nation’s capital on Jan. 22 to urge the agency to fast-track approval of a promising new drug called Eteplirsen to fight Duchenne Muscular Dystrophy, the disease that afflicts their six-year-old son Pietro.
Little Pietro is currently taking part in a clinical trial of Eteplirsen, receiving weekly infusions of the drug, and is already showing signs of significant progress, his father said.
“We’re on week 22 of a 96-week trial. He’s now able to climb stairs. He couldn’t do that before,” Manni Scarso, a broker of private jet sales, told reporters at a press conference in John Paul Jones Park in Bay Ridge on Dec. 4.