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MECP2 Duplication Syndrome – Pediatric Medical Genetics

Sonny Staniscia in a pool

Like all mothers, Sonny Staniscia’s mom Ashley has nothing but wonderful things to say about her little boy, who turned 3 in April.

“He’s a happy little dude,” she says.

Sonny Staniscia in rainbow overallsSonny was born via in vitro fertilization, “so to even get Sonny was a miracle,” says Ashley. He was born in Uniontown, Pa., and when he was a newborn his mom and dad, Steve, had to try different formulas until they found one he could keep down.

As Sonny grew older, he wasn’t gaining weight and suffered from constipation, acid reflux, and hypotonia, or decreased muscle tone. Ashley says that as Sonny graduated to solid foods, he would spit them up and started to show signs of allergic reactions.

His parents took him to an allergist and learned that Sonny is allergic to dairy, peanuts, and eggs. He was having allergic reactions when his parents tried feeding him.

Not only was Sonny not gaining weight, he also was not hitting his developmental milestones. They entered him in Early Intervention, where Sonny received special instruction as well as many other outpatient therapies such as speech, occupational, and physical therapy.

Living in the Moment

Sonny was diagnosed with MECP2 duplication syndrome on May 16, 2022 – “the worst day ever,” says Ashley. MECP2 is the name of a gene that’s involved in the development of neurologic features. The original condition that was discovered is called Rett syndrome, which for a long time the medical community assumed only affected girls. It’s since been learned that a few hundred males also have a change in that gene, and their condition is called MECP2 duplication syndrome. They have long gone undiagnosed simply because medical professionals did not know the condition was possible.

Sonny Staniscia licking a lollipopThe symptoms of MECP2 duplication syndrome are numerous. These boys can have developmental delays, difficulty meeting motor milestones, intellectual disabilities, limited speech, feeding problems, seizures, recurrent infections, and unfortunately, some boys can also have a shortened lifespan, says Leslie Walsh, a genetic counselor at UPMC Children’s. She says that treatment is symptomatic; in other words, we know the list of things these children are at risk for so we can expect they may need things like speech therapy, adaptive devices, and seizure medications. Knowing what these boys are up against allows us to intervene as early as we can for the best possible outcomes. Although there isn’t a cure for this condition, the MECP2 community is very active and engaged in research.

Sonny Staniscia playing with bubblesAshley says one of the scary things about MECP2 duplication syndrome is the spectrum of possible symptoms.

Sonny’s dad, Steve, says, “We need to have a positive attitude about all of this for Sonny.”

Last year, Ashley, who’s an art teacher, had emergency surgery and nearly died. She also was in a bad car accident.

“This forced me as a mom to step back and make him get up and move on his own,” she says. “Sonny’s going to be all right. It’s just a lot. We’re tackling it all.”

With the help of a nurse navigator at Children’s, who schedules multiple appointments for Sonny, he sees specialists in Allergy/Immunology, the Child Development Unit, Ophthalmology, Audiology, the Feeding Clinic, Cardiology, Physical Medicine and Rehabilitation, and Genetics. It was Dr. Shelley Williams in Child Neurology who encouraged the Staniscias to consult with Genetics, which ultimately resulted in Sonny’s diagnosis. He also has had a kidney ultrasound, bloodwork, and recently went had an AAC device evaluation with Speech Therapy.

Ashley is off work this summer since she’s a teacher. She says “I’m in go mode right now. This past year I learned I can do anything. Sonny can do anything.”

“I am going to have as much fun with my buddy while I can,” adds Steve.

Sonny loves music, dancing, and laughing. Baby Shark, Blues’ Clues, and Pete the Kitty are favorites. The Staniscias have lots of family around, and Sonny loves being with his grandparents, cousins, and aunts and uncles, playing in the swimming pool and going to church on Sunday.

Ashley says, “He’s our little ray of sunshine.”