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A Global Prospective Observational Registry of Patients With Pompe Disease

Join a global study on Pompe Disease for better understanding.

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Phase N/A

Pompe disease is a genetic disorder caused by the buildup of a sugar molecule called glycogen in the body's cells. This study observes patients worldwide who have Pompe disease, both in children (infantile-onset) and adults (late-onset). Some patients are on approved treatments, while others are not. The goal is to understand how well treatments work over time, how safe they are, and how they affect patients' quality of life.

Participants must be diagnosed with Pompe disease through specific tests. However, those involved in other experimental treatment studies cannot join. The study will collect data on any bad side effects from treatments and track how patients feel and live with the disease.

  • Study is observational; no new treatment given.
  • Participants can be from anywhere in the world.
  • Data helps improve future treatment and care.
Study details
    Pompe Disease

NCT06121011

Amicus Therapeutics

26 April 2025

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Learn more about clinical trials

What is a clinical trial?

A clinical trial is a study designed to test specific interventions or treatments' effectiveness and safety, paving the way for new, innovative healthcare solutions.

Why should I take part in a clinical trial?

Participating in a clinical trial provides early access to potentially effective treatments and directly contributes to the healthcare advancements that benefit us all.

How long does a clinical trial take place?

The duration of clinical trials varies. Some trials last weeks, some years, depending on the phase and intention of the trial.

Do I get compensated for taking part in clinical trials?

Compensation varies per trial. Some offer payment or reimbursement for time and travel, while others may not.

How safe are clinical trials?

Clinical trials follow strict ethical guidelines and protocols to safeguard participants' health. They are closely monitored and safety reviewed regularly.
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