The arrival on the market of many innovative treatments has been hampered by pharmaceutical companies’ business strategies

Darius is 15 years old and is a dazzling example of how medical research has advanced. Born with a rare and lethal genetic disease, early active cerebral adrenoleukodystrophy — CALD, the same condition that killed his older brother Danuk — the adolescent boy is today, healthy and happy in his hometown of Quintanar de la Orden, in the Spanish province of Toledo. His recovery has been thanks to a gene therapy called Skysona. A benevolent ­virus, a Trojan horse that was introduced into his body when the boy was five years old, it was able to replace the boy’s defective gene with a healthy one, thus saving his life.

But to the frustration of his doctors and other families, Darius’s treatment has not been available on the European market since 2021, due to a decision made by its pharmaceutical company Bluebird Bio that has left dozens of young people in the EU without the opportunity to follow Darius down the same path to survival. “It is difficult to comprehend,” says Carmen Sever, president of the European Lecodystrophies Association in Spain (ELA-Spain), which has helped patient associations to finance and develop the drug.