Doctors examined the little boy and on a subsequent trip, his 11-month-old brother, concluding that the two suffered from mucopolysaccharidosis type VI, a rare genetic disease that has no cure.

The treatment of this rare genetic disease is based on ... in patients are beta thalassemia and two metabolic diseases of childhood, mucopolysaccharidosis type 6 and type 1. In addition, within ...

The treatment of this rare genetic ... two metabolic diseases of childhood, mucopolysaccharidosis type 6 and type 1. In addition, within the Telethon institutes a targeted therapeutic strategy is ...