This rare genetic condition affects approximately 3,000 people in Spain. It causes progressive damage to the nervous system, leading to impaired coordination, fatigue, and speech difficulties. Until now, patients had no therapeutic options to alter the course of the disease.
“"It is a relentless disease for which, until now, we had no alternatives and witnessed the irreversible loss of abilities in our patients."
The oral medication is indicated for patients aged 16 and older. Clinical trials have shown that it effectively slows down neurological decline by activating the Nrf2 protein. Leading hospitals in Barcelona, such as Sant Pau and Sant Joan de Déu, are involved in ongoing research and pediatric trials.
