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07/12/2021

The UCA and Pfizer will study the long-term benefits of an experimental drug that aims to improve the quality of life of children with achondroplasia

A year ago, the Arthroscopic Surgery Unit of Dr. Mikel Sánchez (UCA, according to the Spanish acronym) became the national reference centre for the Pfizer pharmaceutical company´s Phase II multicentre trial on achondroplasia in children. This trial studies a target drug to reverse the effect of the gene mutation that causes this condition. The lead doctors in these trials, Dr Knörr and Dr de Bergua, in collaboration with the pharmaceutical company Pfizer, will now study the long-term benefits of this new drug. The children who began receiving treatment at our centre a year ago will move on to the new extension phase this December.

This experimental drug is essential to treat and improve the quality of life of children that suffer from achondroplasia, a rare disease that causes dwarfism and whose only treatment nowadays consists on the lengthening of extremities. One in 25,000 children suffers from achondroplasia.