Lifeline extended: patients get continued access to rare disease drug
NCT ID NCT05777993
Summary
This study provides continued access to the drug mitapivat for people with pyruvate kinase deficiency who completed earlier research studies. It's for participants who benefited from the drug but can't get it through regular commercial channels. The main goal is to monitor safety while allowing these patients to continue their treatment.
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Contacts and locations
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Locations
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Kyoto Katsura Hospital
Kyoto, 615-8256, Japan
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McMaster Centre for Transfusion Medicine McMaster University
Hamilton, Ontario, L8S3Z5, Canada
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Toho University - Omori Medical Center
Εta-ku, 143-8541, Japan
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Tohoku University Hospital
Sendai, Miyagi, 980-8574, Japan
Conditions
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