Parents of children with Duchenne syndrome and their supporters spend 22nd night outside government office, continuing daily rallies and weekly marches as they say they will not stop until their demands are met.
The parents, representing around 100 children, are calling for access to treatments including Vamorolone, Givinostat, exon-skipping therapies and Elevidys, which are authorized by the US Food and Drug Administration and the European Medicines Agency. They say they have been trying to secure these medicines in Georgia for the past year and a half without success.
The Georgian Ministry of Health says there are still unresolved questions over the safety and effectiveness of the requested medicines, adding that long-term clinical studies have not yet been completed. The Ministry also notes that there is currently no cure for Duchenne muscular dystrophy.
Parents, however, argue the medications are already being used in the United States and Europe and claim the government’s reluctance is largely linked to the high cost of treatment.
Duchenne muscular dystrophy is a rare genetic disorder that causes progressive muscle degeneration, often leading to wheelchair dependence and life-threatening complications.
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Parents appeal to government, Patriarchate over Duchenne treatment access
Health minister: Duchenne medicines do not cure or prolong life
