First patient dosed in phase 1/2 study evaluating SOBI003 for treatment of mucopolysaccharidosis type IIIA (MPS IIIA)
Swedish Orphan Biovitrum AB (publ) (Sobi™) announces that the first patient has been dosed in the phase 1/2 study SOBI003-001. The study is an open-label, non-controlled, multiple-dose study with the objective of assessing the safety, tolerability and efficacy of SOBI003 in nine children aged 1-6 years for the treatment of mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo A syndrome. “Since there is currently no treatment available for MPS IIIA, the initiation of this study is an important first step towards finding a potential treatment for this