New enzyme therapy shows promise for infants with rare muscle disease
NCT ID NCT04910776
First seen Apr 21, 2026 · Last updated May 14, 2026 · Updated 5 times
Summary
This study tests a new enzyme replacement therapy called avalglucosidase alfa in infants with Pompe disease, a rare genetic disorder that causes severe muscle weakness. The treatment is given by IV every other week for up to 4 years. The main goal is to see if more babies survive and can breathe without a breathing machine after one year of treatment.
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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.
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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Locations
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Advanced Medical Genetics- Site Number : 8400002
Hawthorne, New York, 10532, United States
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Children's Hospitals and Clinics of Minnesota- Site Number : 8400008
Minneapolis, Minnesota, 55404, United States
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Cincinnati Children's Hospital Medical Center- Site Number : 8400001
Cincinnati, Ohio, 45229, United States
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Duke University Medical Center- Site Number : 8400004
Durham, North Carolina, 27710, United States
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Investigational Site Number : 0560001
Leuven, 3000, Belgium
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Investigational Site Number : 1560001
Shanghai, 200120, China
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Investigational Site Number : 1560002
Qingdao, 266034, China
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Investigational Site Number : 1580001
Taipei, 100, Taiwan
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Investigational Site Number : 2760001
Bad Oeynhausen, 35392, Germany
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Investigational Site Number : 3800002
Monza, Lombardy, 20052, Italy
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Investigational Site Number : 5280001
Rotterdam, 3015 CE, Netherlands
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Investigational Site Number : 7240001
Esplugues de Llobregat, Catalunya [Cataluña], 08950, Spain
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Investigational Site Number : 8260001
London, London, City of, WC1N 3JH, United Kingdom
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Investigational Site Number : 8260002
Manchester, M13 9WL, United Kingdom
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Seattle Children's Hospital- Site Number : 8400003
Seattle, Washington, 98105, United States
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Stanford Hospital- Site Number : 8400006
Stanford, California, 94305, United States
Conditions
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