Pro-European protests in Tbilisi have amplified calls for state funding for Duchenne muscular dystrophy (DMD) medicines, with demonstrators adopting "Give children medicine" as a key theme. This follows months of regular protests by parents of children with DMD outside the Georgian government administration, demanding access to necessary treatments for the rare genetic disorder.
Former Georgian Health Minister Andria Urushadze stated that treating DMD in Georgia is a matter of "priorities" rather than finances. He estimated that 60-80 individuals in Georgia live with the diagnosis, requiring an annual state budget allocation of 30-35 million lari ($11-13 million) for their treatment. Urushadze suggested that regional procurement could further reduce these costs.
DMD is a severe genetic disorder causing progressive muscle wasting, primarily affecting boys, leading to loss of mobility, respiratory, or cardiac failure. The organisation "Together Against Duchenne Muscular Dystrophy" reports approximately 100 diagnosed children in Georgia, with 1-3 children dying annually due to inadequate medication and treatment.
Urushadze's estimate of 60-80 patients draws on three sources: statistical prevalence (one in 3,500-5,000 live-born boys applied to Georgia's average 20,000 male births annually over a 0-20 year age group), an internal registry maintained by the Duchenne Foundation, and data from the Ministry of Health and Social Protection's rare diseases programme. Such figures are crucial for accurate state budget planning.
The former minister argued that allocating just over 1% of the budget could save dozens of children's lives and prevent severe disability. He emphasised that "it is difficult to remain indifferent to families demanding funding for medicines," framing the issue as a critical test of governmental priorities in public health.
International regulatory bodies have recently approved new treatments, highlighting the evolving landscape of DMD care. The US Food and Drug Administration (FDA) approved Givinostat (Duvyzat) in March 2024, with the European Medicines Agency (EMA) granting marketing authorisation in June 2025 for the first non-steroidal medicine for patients aged six and above.
These advancements provide a concrete pathway for treatment that Georgian patients currently lack. The Ministry of Health could improve planning by establishing a single electronic register of patients, enabling precise budget calculations for an "upper threshold" of 80 patients to ensure no child is left without treatment.