New hope for babies with rare muscle disease when standard treatment fails
NCT ID NCT04327973
Summary
This program provides access to an experimental drug combination (ATB200/AT2221) for children with infantile-onset Pompe disease who are getting worse on their current enzyme replacement therapy. The treatment aims to better control this rare genetic disorder that affects muscles and organs. It's offered to children under 18 who can't join regular clinical trials and whose doctors believe they need different treatment.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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National Taiwan University Hospital
AVAILABLETaipei, 10002, Taiwan
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UCSF Benioff Children's Hospital Oakland
AVAILABLEOakland, California, 94609, United States
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UF Health Shands Hospital
AVAILABLEGainesville, Florida, 32610, United States
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University Hospital of Padova
AVAILABLEPadua, Italy
Conditions
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