For adults with Pompe disease, the medications cipaglucosidase alfa and miglustat are already approved. A new clinical trial now aims to evaluate this combination therapy for use in children aged 0 to 12 years.

This so-called Zip study is focused on children with late-onset Pompe disease. The goal is to assess the efficacy and safety of the combined treatment in this younger age group.

Key eligibility criteria include:

• Age: 0 to 12 years
• Diagnosis: late-onset Pompe disease (Additional criteria apply)

What the study offers:

• Free access to study medication
• Medical care provided as part of the study

Further information is available at clinicaltrials.gov (Study ID: NCT03911505) or directly from the study team.