From left: Nathan and Bennett Falcone, of Erdenheim, smile before participating in the clinical trial sponsored in part by the Progeria Research Foundation and Boston's Children's Hospital earlier this year. (Photo courtesy of the Falcone Family)

From left: Nathan and Bennett Falcone, of Erdenheim, smile before participating in the clinical trial sponsored in part by the Progeria Research Foundation and Boston’s Children’s Hospital earlier this year. (Photo courtesy of the Falcone Family)

by Sue Ann Rybak

Six-year-old Bennett Falcone’s blues eyes sparkle as he sits down at the kitchen table to eat tortilla chips with melted cheese. When he is asked how they are, he replies with a thumbs up. His brother Nathan, 10, sits next to him nibbling on chicken nuggets.

“Daddy, can I have some water,” Nathan asks.

“Sure,” his father Mark replies.

As Bennett knobby fingers reach for his plastic Lego movie cup, his father asks “What was your favorite thing to do this summer?”

“Was it the Spy Museum, the Outer Banks or the tractor pull,” his mother, Phyllis Falcone, asks.

“Everything,” Nathan exclaims.

It’s a typical end of the summer conversation except for one thing.

Both Nathan and Bennett have an extremely rare and severe type of progeria called mandibuloacral dysplasia type B. In Fact, Nathan and Bennett are two of only three children in the United States with this rare form of progeria. Also known as Hutchinson-Guilford progeria syndrome, progeria is a fatal genetic disease which causes rapid aging in children. Children with classic progeria usually die at an average of 13 from heart attack or stroke.

With Nathan recently turning 10, it’s a race against time to find a cure.

That’s why the Falcones are once again hosting their fifth annual Make a Splash fundraiser on Sunday, Sept. 13 from 11 a.m. to 3 p.m. at the Flourtown Country Club, 150 McCloskey Road in Flourtown. All proceeds benefit the Progeria Research Foundation.

The family-friendly event includes swimming, dancing, a chance to win Flyers tickets (in the Mayor’s Box), Eagles tickets, NASCAR tickets, a Playstation and more.

Phyllis believes miracles happen every day and a cure for progeria could be on the horizon.

In 2012, a study found that a farnesyltransferase inhibitor (FTI) called lonafarnib, a drug originally developed to treat cancer, improved children with Progeria’s rate of weight gain, bone structure, and, most importantly, vascular stiffness, a predictor of heart attack and stroke. In 2014, further studies revealed that lonafarnib can extend the lives of children with progeria.

“Lonafarnib is giving children with progeria stronger hearts and longer lives,” said Leslie Gordon, MD, PhD, and medical director for the Progeria Research Foundation.

The Progeria Research Foundation recently expanded the trial. Thanks to generous donors, 70 children from 30 countries have enrolled to date. However, donations are still desperately needed to maintain the funding for the clinical trial.

Gordon estimated that the current clinical trial will cost $2.8 million. She said to date PRF has raised $2.2 million.

Phyllis said thanks to the Progeria Research Foundation the disease has gone from total obscurity to a treatment in less than 15 years.

“Although we will not rest until a cure is found for Progeria,” she said, “we are grateful for any improvements gained through treatment. A little more hair on their heads, stronger muscles, less aches and pains, a little taller, improved flexibility, all of these improvements help them enjoy their lives now.”

For more information or to buy tickets for the fundraiser go to www.facebook.com/events/944529995585774/ or interland3.donorperfect.net/weblink/weblink.aspx?name=E13407&id=24. Tickets will also be available at the door.

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