3 results
The objective of the study is to asses the efficacy and safety of intravenous ATB200 co-administration with oral AT2221 by evaluating the changes in key clinical outcome measures (eg, motor, respiratory, fatigue) in adult subjects with late-onset…
The objective of the study is to gather more information about the long-term effect of enzyme therapy in patients with Pompe disease with a different severity of disease. The goal is also to set guideline for start and stop of treatment and to…
The overarching goal is to optimise the treatment of KD in children/adolescents across Europe.KD-CAAP will test the hypothesis that adding immediate adjunctive corticosteroid treatment to IVIGand aspirin will reduce CAA rates in unselected KD…