Children Waiting While Time Runs Out: The Human Cost of Duchenne in Georgia

For the families of children living with Duchenne Muscular Dystrophy (DMD) in Georgia, time is no longer measured in birthdays, school years, or holidays. It is measured in lost movements. In the ability to climb a staircase. In the strength needed to hold a glass of water. In the realization that every month without treatment is another month the disease quietly advances.

Duchenne is a rare and progressive genetic disease that gradually destroys muscle tissue. Children affected by it slowly lose the ability to walk, move their arms, breathe independently, and eventually survive without intensive medical support. For the parents who have organized the largest patient advocacy movement in recent Georgian memory, the disease did not simply alter their lives. It divided them into two realities: before the diagnosis and after the diagnosis.

About 100 children are currently registered in Georgia with the condition. (Civil Georgia coverage of Duchenne families meeting with PM Kobakhidze)

A diagnosis that rewrites the family

“I think I was the first one to notice something was wrong,” said one father, whose son was diagnosed at one and a half years old. “He was struggling to balance. Since then, I have been continuously fighting.”

The pattern is familiar to many DMD families. A toddler who walks late, falls often, struggles to climb stairs, or has unusually large calf muscles. Bloodwork shows extremely elevated creatine kinase, the enzyme that leaks out of damaged muscle. Genetic testing confirms a mutation in the dystrophin gene. The future the family had imagined collapses into a different one.

Unlike many diseases where recovery is possible, Duchenne takes away abilities permanently. Once a child loses the ability to walk or move independently, those milestones rarely return.

We do not have much time. Every day, every week, every month, the children are getting worse. That is why the parents are stressed every single day.

What deterioration looks like at home

Parents describe a painful reality shared by nearly every family affected by DMD: watching deterioration happen in slow motion. One child is beginning to struggle climbing stairs. Another is already losing mobility in his hands. Many are dependent on glucocorticoids that help delay progression but create severe side effects, including weight gain, growth problems, weakened bones, and hormonal complications.

“We are struggling with two difficult choices,” one father explained. “If we reduce the steroids, they become less effective. If we increase them, the side effects become worse.”

The emotional burden extends beyond medicine. Parents must explain a devastating diagnosis to relatives while simultaneously trying to protect their children from the psychological weight of the disease. One father said his teenage son understands more than he openly admits.

“He does not like to talk about the disease,” the father said. “He enjoys movies, jokes, playing games, driving his scooter outside. He has his own happy world, and I do not want to disturb it.”

When parents become activists

For a year, parents in Georgia urged the authorities to allow the import of costly medicines used internationally. In January 2025, they founded the organization Together Against Duchenne Muscular Dystrophy. (JAMnews, We have very little time)

The advocacy intensified after the death of 17-year-old Avtandil Bregadze in early March 2026. He was buried on March 3. By April 20, parents had brought blankets and beanbags to the government administration building in Tbilisi and begun an open-ended protest. (JAMnews)

Thousands of supporters now participate in regular marches. They bring food, donations, wheelchairs, and emotional support to affected families. The movement has also attracted the attention of religious leaders, public organizations, and representatives connected to the World Health Organization. The Catholicos-Patriarch of All Georgia, Shio III, met with the protesters and agreed to mediate with the government. (Georgia Today, Patriarchate mediation)

Still, for families, progress feels painfully slow.

”Treatment exists somewhere else, but not for your child”

The core frustration is simple: treatments already exist. Parents point to therapies approved by the United States Food and Drug Administration and the European Medicines Agency and ask why Georgian children remain without access.

“What hurts the most,” one father said, “is knowing that treatment exists somewhere else, but not for your child.”

One father described a Georgian family split between two countries. One child lives in the United States and received access to therapy. The other remained in Georgia.

“The difference between them is enormous,” the father said.

The emotional impact of that comparison is impossible to ignore. Parents interviewed for this article repeatedly described the same dilemma: whether to leave Georgia in search of treatment or remain and continue fighting publicly for change. Some families already attempted to move abroad. Others returned home after struggling to navigate foreign healthcare systems.

One father, who speaks seven languages, said friends and relatives constantly encourage him to emigrate. Instead, he chose to stay.

“I prefer to stay in Georgia and struggle,” he said. “If I leave, then I am giving up this fight.”

The cost of being heard

That struggle has transformed ordinary parents into activists. Many who once avoided public attention now speak openly on television, participate in protests, and organize demonstrations almost daily.

“My wife and mother were very stressed initially,” one father explained. “Now they understand publicity is necessary if we want results.”

The families insist their demonstrations are peaceful and lawful. They also believe public pressure became necessary because negotiations alone failed to produce meaningful progress. “We can protest and negotiate at the same time,” one father said. “There is no contradiction.”

For these parents, the fight is no longer only about their own children. “This struggle will affect future generations,” one father said. “Not only Duchenne patients, but all children suffering from rare diseases.”

Public pressure has produced movement. According to parents, authorities have begun contacting patients and conducting broader clinical assessments, and Health Ministry consultations have expanded. The families also report that misinformation has complicated the public debate. An investigation by Myth Detector documented 75 fake social media profiles, some apparently AI-generated, amplifying narratives that DMD treatments are unsafe or ineffective. (Myth Detector investigation on AI-generated trolls)

Hope, in the simplest form

Even amid exhaustion and uncertainty, the families continue to speak with remarkable hope. They believe their movement will eventually succeed. Not because the fight is easy, but because they feel they no longer have any alternative.

And in the eyes of parents watching time slowly steal their children’s futures, silence is no longer an option.

For background, read reading health information about DMD carefully and Two Mothers, Two Realities.

Disclaimer: This article is a reported piece based on public sources. It is informational and does not constitute medical advice. Decisions about diagnosis or treatment must be made with a qualified care team.