Yes from Aifa to the first drug for cancers caused by a rare disease in children. Selumetinib shrinks the size of inoperable tumors in children, reducing pain and improving quality of life.
NF1 is a debilitating genetic condition which affects one in 3,000 people worldwide and which in Italy is estimated to affect around 20,000. In between 30 and 50% of patients, tumors develop on the nerve sheaths (plexiform neurofibromas) causing problems such as disfigurement.