'Give our kids the medicine': Georgian parents are sleeping in the rain for the right to keep their sons alive

Around 100 boys in Georgia are living with Duchenne muscular dystrophy - a disease that has never allowed a child to grow old. Their parents have spent weeks protesting outside government offices, saying their sons cannot wait any longer for treatment.

“Give our kids the medicine. What if it was your child?”

Those words - scrawled on banners and shouted into the cold Tbilisi wind - have become the rallying cry of Georgian parents camped outside the country’s Government Chancellery since 20 April. Day and night, through rain, bitter winds and near-freezing temperatures, they have demanded access to medicines that could slow a disease that is, without exception, fatal.

They are not asking for a cure. There is no cure. They are asking for a chance to slow the progression of a condition that destroys children’s bodies year by year.

Duchenne muscular dystrophy (DMD) is a rare genetic disorder that destroys muscle tissue from birth. It affects almost exclusively boys because the faulty gene sits on the X chromosome, and it progresses with relentless efficiency. By their teenage years, most boys with Duchenne require a wheelchair. Later, their hearts and lungs begin to fail. Average life expectancy is typically between 20 and 30 years.

In Georgia - a country of fewer than four million people on the edge of the Caucasus - around 100 children are living with the diagnosis. Anewz has spoken to several of their parents, as well as the organisation leading the campaign, over recent weeks. Their accounts paint a picture of families exhausted by grief and driven by anger as they watch their sons deteriorate while political and bureaucratic deadlock continues.

“Every passing year means that their life is being taken away. Neither birthdays nor New Year - nothing makes us happy anymore.”

A disease that steals childhood, year by year

Tako Gogoladze, the mother of nine-year-old Andria Tevzadze, has attended almost every night of the protest. Speaking to Anewz, she described the reality of living with Duchenne.

“From about the age of 10, children are confined to a wheelchair,” she said. “Then internal pathologies begin - the heart and lungs begin to degenerate, they lose strength in their hands and limbs. Life expectancy is about 18 to 20 years.”

Kakha Tsikarishvili, father of 13-year-old Nikoloz, described the disease in similarly stark terms.

“Duchenne’s disease actually deprives children of their childhood,” he told Anewz. “From about seven years old, it becomes difficult for a child to climb stairs. By 11 or 12, he is in a wheelchair. By 17 or 18, even breathing becomes difficult. By the age of 20, they can only move their fingers.”

Without medical intervention, he said, a fatal outcome before the age of 30 is almost inevitable.

In the past 18 months alone, three Georgian boys with Duchenne have died - aged 17, 22 and 27.

The treatments at the centre of the dispute

For the first time in the history of Duchenne, treatments now exist that can significantly slow the disease’s progression. They cannot cure or reverse it, but families say they could buy children precious years.

Parents are demanding that the Georgian government fund and import four therapies, all of which have been authorised by major international regulators.

Vamorolone (Agamree)

A next-generation anti-inflammatory steroid with significantly fewer side effects than older steroids currently used in Georgia. It was approved by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) in late 2023, and by the NHS in the UK in December 2024. It is already funded in countries including Germany, Austria and the UK.

Givinostat (Duvyzat)

The first non-steroidal Duchenne treatment, approved by the FDA in March 2024 and by the EMA in June 2025. Taken daily as a pill or syrup, it reduces muscle inflammation and slows muscle deterioration. It is already in use in Italy, Germany and other countries, while Georgian children living abroad are already receiving it.

Exon-skipping therapies 

These gene-targeted treatments help the body produce a partially functional form of the dystrophin protein, slowing disease progression. Several versions have been authorised by the FDA and are already in use in the U.S. and Japan. Three are registered in Georgia but have never been imported.

Elevidys (gene therapy)

A one-time intravenous gene therapy designed to deliver a functioning gene to muscle cells. It is also the most expensive option, costing around $3 million per patient. The therapy received FDA authorisation in 2023 and 2024 but has not been approved in Europe. It remains under clinical monitoring following rare but serious adverse events and is currently restricted to ambulant patients.

Zakro Gvishiani, chair of the organisation Let’s Fight Duchenne Muscular Dystrophy Together, told Anewz that even limited improvements can transform a child’s life.

“We have a Georgian child with American citizenship who has undergone gene therapy,” he said. “Until recently, he could not even manage one floor of stairs. Now he can handle nine floors independently.”

Government concerns over safety and effectiveness

Parents insist they are not asking the government to take unreasonable risks. They argue that the FDA and EMA — widely regarded as two of the world’s strictest medicines regulators - have already carried out extensive assessments of the drugs.

International pharmacoeconomics expert Tiniko Turdziladze, speaking to a Georgian media outlet, said regulators weigh risks differently in rare diseases such as Duchenne because the disease itself is so devastating.

“The concept is to assess the risk of the disease and the benefit of the drug,” she said. “In the case of Duchenne, the risk is very high - therefore, drugs are released with less data, and observation continues.”

“Research is underway globally and this disease may actually be cured in a few years. That is why it is so important for us to take these medications today — so that our children are still alive to receive whatever comes next.”

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The Georgian government has acknowledged that Duchenne is a healthcare priority, but says questions remain over the safety and effectiveness of the medicines parents are demanding.

This part one of a three part series about Duchenne treatment access in Georgia. In part two, Nini Nikoleishvili speaks to more campaigning families and looks at why the situation in Georgia differs to several other European countries. 

"When we return home, the children ask us: will there be medicine soon? Will we be okay? Can I run like all the other children?" 

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