Six-year-old Pietro Scarso continues to fight, and with the help of new treatment, the Bay Ridge youngster has made significant strides that his family and supporters hope will help him knock out his illness for good.
For years, Pietro has battled Duchenne Muscular Dystrophy (DMD), an inherited disorder of progressive muscular weakness, typically in boys.
During a conference at John Paul Jones Park on Friday, December 4 to provide details about the second annual PJs for PJs, a pajama party event that is a community-wide fundraiser, the Scarso family — Pietro’s mother, Dayna, father, Manni and brother, Niko — joined State Senator Marty Golden in discussing Eteplirsen, a drug that Pietro has been taking since this past summer to treat DMD, and their efforts to get it approved by the Federal Drug Administration (FDA).
“Within a few weeks of treatment, Pietro started doing things that he’s never done before, such as jump off the ground with two feet,” Manni said, adding that before treatment, he would tire very early while playing. “He’s able to climb the stairs. He still labors but he can do it. His stamina has increased dramatically.”
“His confidence has grown a lot since the treatment,” added Dayna. “It works. He couldn’t even lift his arms straight up. Now he’s lifting his arms, climbing ladders. There are no side effects. We need the FDA to approve this.”
With an FDA Advisory Committee meeting on the matter tentatively set for Friday, January 22 2016 in Washington D.C., the Scarso family plans to attend along with others to discuss the success the drug has had with Pietro.
“We’re just going to tell his story. Before the trial, he couldn’t walk up the steps without a lot of labor. Now he can do three or four steps without holding on,” said Dayna. “He’s walking long distances and not getting tired. He went to Disney and did the entire park fine.”
The goal, said Golden, is to get FDA approval as early as February 2016. “If we can do that, it can change the lives of so many kids and families,” he stressed. “We need to push this forward.”
According to Manni, if approved, Eteplirsen would be the first drug ever approved for DMD.
“Thirty days after the meeting, they will rule on whether the drug gets approved or not and whether they are going to require additional data,” he said, estimating that by the end of February, there will be an answer as to whether or not the drug will get the green light. “Hopefully, it will be approved because there are some kids that can get the drug, but the trial is so limited.”
The second annual Pjs for PJ will take place on Thursday, December 17. For more information, visit www.pietrosfight.org.