Dyker Heights parents praise FDA approval of muscular dystrophy drug

Scarsos say Eteplirsen is helping their son

September 23, 2016 By Paula Katinas Brooklyn Daily Eagle

Little Pietro Scarso started his transfusion treatments with the experimental drug last year. Photo courtesy of www.pietrosfight.org

Share this:

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.

Eteplirsen is manufactured by the pharmaceutical company Sarepta Therapeutics.

Duchenne Muscular Dystrophy is a rare genetic disorder characterized by progressive muscle deterioration and weakness. The disease affects one in every 3,500 boys in the U.S.

Approximately 400-600 males are born with are born with Duchenne Muscular Dystrophy in the U.S. each year.

Most patients wind up wheelchair-bound by the time they reach their teens, and many patients die before they reach the age of 30.

The Scarsos released a statement through the nonprofit organization they founded, Pietro’s Fight, to express their gratitude for the turn of events. Manni is a broker of private jet sales. Dayna is a stay-at-home mother who has devoted countless hours caring for her son and raising funds for Duchenne Muscular Dystrophy research.

“We want to thank every single person who has supported Pietro’s Fight over the past four years. With the community, business and our neighbors, we have raised more than $1 million for research and our efforts and advocacy have made a difference. The boys in the trials such as our son Pietro, in his 62nd week, are walking longer, getting in and out of their beds, they are stronger, and can so much more than they would be able to do without this drug,” the statement reads.

Samantha Altilio, executive director of Pietro’s Fight, also issued a statement. “The FDA did their job in making sure that Eteplirsen was a safe drug prescribed to these children. It took 115 days for the FDA to come to a decision, it took courage from these boys and their parents, and it took support from the community and the nation to get this achieved,” she stated.

State Sen. Marty Golden (R-C-Bay Ridge-Southwest Brooklyn), who befriended the Scarsos and assisted in fundraising efforts, said he was delighted when he learned of the FDA decision.

“We have been waiting for this day for a long time, when the boys without the Exon 51 gene can get the treatment they need in the fight against Duchenne Muscular Dystrophy. I salute the Scarso Family and all you have done over the past four years, and I am proud to have been a partner with you in this mission. This community has and will continue to embrace and stand with Pietro’s Fight and the DMD community to further advance research and raise awareness,” Golden said.

Pietro’s Fight hosts five main fundraisers events throughout the year. “Fight Night,” an adult-only gala, is held every March at the El Caribe Country Club in Mill Basin. “Bump Crash Smash,” a kid-friendly party, is held at an amusement park every spring. There is also “Pietro’s Fight to the Finish Line,” a 5K run/walk every September; an adult costume party at or near Halloween at Cebu Restaurant in Bay Ridge; and “PJs for PJ,” a community-wide in which schools and businesses donate funds and wear there PJs for Pietro Joseph.

For more information, visit www.pietrosfight.org.