Dani, a two-year-old boy from Málaga, has made history by becoming the first patient under three years old to receive experimental gene therapy for hereditary spastic paraplegia type 50 (SPG50) in Spain. This genetic neurodegenerative disease, which affects about one hundred people worldwide, causes severe delays in motor and cognitive development, and can also lead to a possible loss of mobility. The treatment was possible thanks to the collaboration between the Hospital Sant Joan de Déu in Barcelona and the Columbus Foundation of Valencia, entities that work to offer new hopes to children with ultra-rare diseases in the country.
On September 9, Dani received a dose of the drug Melpida, the only gene therapy for SPG50, administered without complications. This innovative treatment has been the result of the interaction among several institutions and experts, with an international support network that includes the development of the drug in the United States. and the production by a Spanish biotechnology company. Dani's experience reflects Spain's potential to provide access to advanced treatments without families having to travel abroad, highlighting the importance of altruistic cooperation in the care of rare diseases. Its evolution will be closely monitored, with the expectation of seeing significant improvements in its condition in the coming months.
Read the full news article on 20minutos.
