Meet Tas
A brave 10-year-old boy fighting Duchenne Muscular Dystrophy with courage and determination.
A brave 10-year-old boy fighting Duchenne Muscular Dystrophy with courage and determination.
The Sebestyén family from Transylvania is fighting a battle against time. Their son Tas was a healthy and energetic child until the age of 5, when his parents, Gabriella and Béla, began noticing that he had difficulty climbing stairs, getting up from the floor, and keeping up with other children his age.
Shortly after, the kindergarten teachers also reported the problem, so they immediately turned to a pediatrician who referred them to a neurologist. The medical examinations were performed in Sepsiszentgyörgy, and quickly confirmed their suspicions.
The blood test revealed that Tas's CK (creatine kinase) level had reached 40,000 – this value typically ranges between 200-300 in a healthy man. This was a clear sign of muscle atrophy. Two months later, the genetic test confirmed: Tas suffers from Duchenne Muscular Dystrophy.
Despite the challenges he faces, Tas remains a cheerful, smiling, and humorous boy. He enjoys playing with Lego, video games, and swimming when possible. His courage and resilience in the face of DMD inspire everyone around him.
Duchenne Muscular Dystrophy (DMD) is a genetic disease that primarily affects male children. It is estimated that one in 3,500 newborn boys is affected. The problem is caused by the absence or faulty production of a protein called dystrophin, which would help in healing even the smallest injuries to our muscles.
DMD primarily affects the skeletal and cardiac muscles, leading to difficulty with movement, breathing, and heart function. The disease typically progresses in the following stages:
Parents notice the symptoms of DMD disease in toddlerhood, as this is when the proximal muscles begin to weaken, and the child has movement difficulties. As they grow, it becomes noticeable that they run slower than their peers, and around the age of 6, climbing stairs becomes increasingly difficult, and the child frequently falls.
Most DMD patients are confined to a wheelchair by the age of 10-12, and the disease eventually reaches the muscles of internal organs after the limbs. The expected age limit is 26-28 years, but there are known cases of young people who have reached the age of 30.
While there is currently no cure for DMD, various treatments can help manage symptoms and improve quality of life:
Tas was born healthy and showed normal development during his first years of life.
At age 5, parents notice Tas having difficulty climbing stairs and getting up from the floor. Kindergarten teachers also report concerns about his movement.
Pediatrician refers Tas to a neurologist in Sepsiszentgyörgy after observing muscle weakness.
Tests reveal Tas's CK (creatine kinase) level had reached 40,000, compared to the normal range of 200-300, indicating muscle atrophy.
Genetic testing confirms that Tas suffers from Duchenne Muscular Dystrophy. The family begins to learn about the condition and treatment options.
Tas begins taking Deflazacort, a steroid imported from Germany, as Romania has not yet approved it. He also starts regular therapy sessions.
Tas attends therapy daily: three times a week for kinesiotherapy, once for movement therapy, and once for therapeutic massage.
The family is raising funds for Elevedys, a gene therapy product approved in June 2024 that could delay the destruction of muscle cells and significantly improve Tas's quality of life.
Tas needs access to Elevedys, a gene therapy product that costs $3.5 million. This treatment could delay the destruction of muscle cells and significantly improve his quality of life.
The family currently lives on almost the top floor of a ten-story apartment building. They need to adapt their apartment to Tas's needs or ideally move to a house that would be more accessible.
Tas requires continued access to steroids imported from Germany, as well as daily therapy sessions, which cost the family an entire salary each month.
As Tas's condition progresses, he will need a wheelchair, patient lift, and other adaptive equipment to maintain his independence and quality of life.
Tas is fortunate to have a loving and dedicated family supporting him through his journey with DMD. His parents, Gabriella and Béla, have become fierce advocates for his care, navigating the complex healthcare system and researching the latest treatment options.
His little sister, Panna, also needs her parents' attention and care. The family tries to raise their children according to the same principles and rules as in an average family, ensuring that both children are happy despite the challenges they face.
The family has also connected with another family in Árapatak whose child was also diagnosed with DMD. They maintain constant contact, helping each other through this difficult journey.
Despite the daily struggles, the family has learned to enjoy the little things and live consciously. As Gabriella says, "We plan ahead, but we don't stress about it every day, because if we did, we would go crazy. We always deal with current issues, and I feel that perhaps we wouldn't live so consciously if we didn't have to struggle with this."
Every day is a challenge for Tas. The simple movements that other children do easily are extremely difficult for him:
Taking steroids – although it's the only thing that helps preserve Tas's muscle integrity – also comes with severe side effects:
School has also presented challenges. Tas has had to change schools multiple times due to accessibility issues. When he was first enrolled, his class was on the second floor. After a year, they had to find a ground-floor class because his parents could no longer carry him up the stairs. They had to transfer again when the school started renovations and couldn't provide a ground-floor room.
Your support can make a significant difference in Tas's life and his fight against Duchenne Muscular Dystrophy.