By Fiona Slusarev
September is Muscular Dystrophy Awareness Month, a time that highlights how individuals can be affected by any of the multitude of types and variants of this genetic muscle-weakening condition. For one Wellington family, living with limb-girdle muscular dystrophy type 2C (LGMD2C) has become a daily reality.
Wellington resident Sheena Urdaz is raising two resilient girls, ages 10 and 13, who are fighting against the clock and LGMD2C. Urdaz is doing all she can to raise awareness and keep this exceptionally rare disease from stealing her daughters’ abilities to run, jump and play.
Urdaz’s girls are two bright and chatty kids who love art and animals. The youngest is drawn to the performing arts, playing the violin, recorder and piano, and also sings. She is reportedly an animal whisperer, owns an axolotl and wants to be a veterinarian when she grows up. Her older sister is accomplished in the visual arts, sewing and creating all sorts of crafts, masks and clothes. She loves her family’s dogs and two cats, plays the French horn, and wants to work in the medical field when she’s older.
Urdaz herself is in the medical field as a physician associate, which is why, four years ago, she noticed something was wrong with her eldest after returning from gymnastics camp. Urdaz took her to the clinic where her daughter was found to have elevated creatine kinase (CK) and be in rhabdomyolysis, a life-threatening condition where muscles break down. She was hospitalized for four days.
After recovering with no recurrent symptoms, doctors told Urdaz not to worry and to get her daughter checked again later. In 2024, Urdaz requested the pediatrician check her daughter’s CK levels again, which came back high. Through a litany of tests and specialists — a rheumatologist, gastroenterologist and geneticist — her eldest daughter’s seemingly innocuous and unrelated history of toe-walking, recurrent falls and frequent breaks during exercise came together.
“We got my oldest one’s results back in April 2025 when I was at Universal on spring break with them,” Urdaz said. “It was devastating. I’m standing in the park, [with] my kids on a ride, and I’m crying getting the results because I knew what it meant.”
Within two weeks, her younger daughter was also tested and likewise diagnosed. The sisters joined the estimated 5,000 other individuals diagnosed with LGMD in the U.S., according to the Muscular Dystrophy Association.
LGMD2C, like all types of muscular dystrophy, is progressive, but in LGMD the muscles that are weakening and atrophying are concentrated around the shoulders and hips. It is attributed to mutations in the sarcoglycanopathy (SCG) gene, which is associated with abnormalities in the proteins inside muscle fibers needed for muscle function.
Individuals suffering from this condition can have symptoms like a swaying-walk, toe-walking, enlarged calf muscles, curvature of the spine, joint contracture, restricted mobility, or potentially fatal cardiac and respiratory complications. Many become wheel-chair bound as teens.
While there is currently no cure, Urdaz is on a mission to raise awareness about LGMD and help fund research trials. Since 2025, she has served on the board of CureSCG, a nonprofit that is dedicated to finding a cure for LGMD by bringing together researchers, clinicians, patients and families, while advancing and funding research. The organization was founded in 2024 by a family in Dubai, whose daughter has LGMD, when they realized no such organization existed.
“Whenever I can get away, I try to go to conferences,” Urdaz explained. “I’m trying to get the word out, trying to get the [CureSCG] name out there and trying to get more people on board and funding.”
Urdaz is fighting to reopen a UF Gainesville Powell Gene Therapy Center trial ATA-200 with principal investigator Dr. Barry Byrne. CureSCG and Urdaz are trying to raise $2.5 million by the end of November to reopen this trial, which has already shown measurable improvement in the four children treated in the first phase of the trial.
This trial, originally brought to the U.S from France by the Dion Foundation, works by delivering a copy of a working SCG gene that children with LGMD are missing, in order to slow or prevent the atrophy of their muscles. This can only be done in a limited time frame, because once muscles are gone, there is no way to rebuild them. The study is limited to children 13 and younger, making this the last eligible year for Urdaz’s eldest daughter.
Should the study reopen, Urdaz hopes her oldest daughter might be selected for one of the four spots that can change the trajectory of her life. While her oldest can’t stand up from the floor without assistance and her youngest can’t climb stairs and is often carried, this superhero single mom is doing everything she can to keep her daughters mobile, from physical therapy to driving across the state for medical appointments.
On Saturday, Sept. 26, Urdaz is running a virtual 5K through the LGMD Awareness Foundation, and on Wednesday, Sept. 30, LGMD Awareness Day, Wellington Elementary School is hosting a wear green spirit day to raise awareness.
“A disease like this can land in any family; it doesn’t ask permission. Hope isn’t naive here. There is real science moving forward in real children, and we need more people to jump on board and push this forward,” Urdaz stressed. “We’re not powerless in this: a $10 registration, a shared post, a donation to CureSCG, just showing up and showing you care — every one of those is a real thread.”
To explore CureSCG or make a tax-deductible donation toward the ATA-200 trial, visit www.curescg.org. To support the vital ATA-200 study through Urdaz, visit www.gofund.me/c86bf6bd4.
Run with Urdaz on Saturday, Sept. 26 at 8 a.m. at Scott’s Place Playground, registering for your own Girdie 5K at www.lgmd-info.org/pl/5K. You can further support Urdaz and her family on Instagram @raisinglgmd.
