Marketed as Skyclarys by Biogen, the drug represents a historic milestone for patients suffering from this condition, which typically appears during adolescence. While it is not a cure, the treatment is designed to slow down the progression of physical decline and neurological symptoms.
“"Until now there was nothing, so this is hopeful. Going to the doctor and being told there is a drug is a lot."
Scientific efforts are also concentrated in Badalona, where the biotech firm Biointaxis—a spin-off from the Germans Trias i Pujol Research Institute (IGTP)—is developing a promising gene therapy. Led by Dr. Antoni Matilla Dueñas, the research aims to restore the production of the frataxin protein, addressing the root cause of the disease.
