This is the third and final article in AnewZ’s series examining the fight for access to treatment for children with Duchenne muscular dystrophy in Georgia, and the irreversible human cost of delay.
This is arguably the most important and least discussed aspect of the entire debate. Duchenne is not a disease in which waiting a year or two while regulators deliberate causes inconvenience. It causes permanent, irreversible physical damage.
Muscle tissue lost to DMD does not regenerate. A child who loses the ability to walk at 11 because access to treatment was delayed will not regain that ability once the drugs arrive at 13. The window for intervention is narrow - and, for each of these children, it is closing every day.
“Every day, every month, every year of government inaction has deadly consequences,” Tsikarishvili said. “It is precisely because of this inaction that we have lost three children in the last year and a half.”
The families are also thinking beyond their own sons. Speaking to Anewz, Gvishiani stressed that the campaign is not only for children who have already been diagnosed.
“We are fighting for those children who may be diagnosed in the future, and for their parents who will have to go through this difficult path,” Tako Gogoladze said. “When there was no medicine, parents did not ask for it. But today, thank God, there is medicine - and we are not going to give up.”
The parents’ organisation has secured an agreement with Georgia’s Public Broadcaster for a live broadcast, likely to take place in the coming days, during which they hope to be joined by medical experts and - if the ministry agrees - a government representative. Whether that dialogue will produce movement remains to be seen.
If it does not, the families say they are prepared to escalate the matter. Tsikarishvili mentioned the possibility of bringing a case before the European Court of Human Rights in Strasbourg, arguing that the state’s inaction constitutes a violation of the right to life under Article 2 of the European Convention on Human Rights - a serious and credible legal avenue, given existing precedents surrounding state obligations in healthcare.
For now, however, the focus remains on a square in Tbilisi, where parents are spending another night on wooden benches in the rain, while their sons wait at home for news. The banners express what every parent in that square feels every waking hour.
“Give our kids the medicine.”
“What if it was your child?”
Vamorolone: Funded in Germany, Austria and the UK through the NHS. At the final review stage in several other countries.
Givinostat: Funded in Italy, Spain and Scotland. Under active consideration in the UK, Ireland and Germany. A regional distribution agreement covering Central and Eastern Europe - including Poland, Romania, the Czech Republic, Hungary and Bulgaria - has been signed, laying the groundwork for future procurement.
Exon-skipping therapies: Actively used in the U.S. and Japan. European-wide approval is pending, while clinical trials remain ongoing.
Elevidys (gene therapy): Available in the U.S. Not approved in Europe. Clinical monitoring remains ongoing following rare serious adverse events. Currently restricted to patients who are still able to walk.
AnewZ spoke directly with parents of children with Duchenne muscular dystrophy in Tbilisi, Georgia, and with the chairman of the parents' campaign organisation.
The Health Minister’s statements were drawn from his parliamentary address on 29 April and a public briefing on 18 April. Background medical and regulatory information was verified against publicly available FDA, EMA and NICE documentation.
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