3 results
The objective of this extension study is to assess the long-term safety and efficacy of Myozyme treatment in patients with Late-Onset Pompe Disease who were previously treated under the placebo-controlled, double-blind study AGLU02704.
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…
Dose Escalation Objectives:Primary:* Determine maximum tolerated dose (MTD) and RP2DSecondary:* Establish tolerability of epcoritamab* Establish pharmacokinetic (PK) profile after single and multiple doses* Evaluate immunogenicity* Evaluate anti-…