Dyker Heights parents praise FDA approval of muscular dystrophy drug
Scarsos say Eteplirsen is helping their son
The dedication and determination of Dyker Heights parents who fought for federal approval of an experimental drug to treat their son’s rare disease has paid off. The U.S. Food and Drug Administration (FDA) recently announced that it has approved the use of Eteplirsen to treat Duchenne Muscular Dystrophy.
Manni and Dayna Scarso had been advocating for the approval for more than a year. The couple testified at FDA hearings, organized petition drives and wrote letters to lawmakers urging that the potentially life-saving drug, also known as Exondys 51, be approved for widespread use across the country.
Their son, Pietro Joseph Scarso, who was diagnosed with Duchenne Muscular Dystrophy at the age of 3, is undergoing a series of experimental infusion treatments with Eteplirsen that began in 2015 when he was 6 years old.
The infusion treatments are designed to supplement an absent gene in the body, Exon 51, and slow down the progression of the devastating disease.