New hope for infants with pompe disease: experimental drug now available

NCT ID NCT04327973

AVAILABLE Disease control Sponsor: Amicus Therapeutics Source: ClinicalTrials.gov ↗

First seen Nov 01, 2025 · Last updated May 16, 2026 · Updated 24 times

Summary

This program offers early access to an experimental drug (ATB200/AT2221) for children with infantile-onset Pompe disease who are not getting better with standard treatment. It is for kids up to 18 years old who cannot join other clinical trials. The goal is to provide the drug while researchers continue to study its effects.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

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Study contacts

Contact

Phone: •••-•••-•••• Email: •••••@•••••
Contact

Phone: •••-•••-•••• Email: •••••@•••••

Locations

National Taiwan University Hospital
AVAILABLE

Taipei, 10002, Taiwan
UCSF Benioff Children's Hospital Oakland
AVAILABLE

Oakland, California, 94609, United States
UF Health Shands Hospital
AVAILABLE

Gainesville, Florida, 32610, United States
University Hospital of Padova
AVAILABLE

Padova, Italy

Conditions

Explore the condition pages connected to this study.

POMPE DISEASE INFANTILE-ONSET