New pill offers hope for patients with rare, Treatment-Resistant blood disease
NCT ID NCT05828108
Summary
This study tested a new oral drug called bitopertin for adults with Diamond-Blackfan anemia (DBA) who don't respond well to steroids. DBA is a rare, inherited condition where the bone marrow fails to make enough red blood cells, often requiring frequent transfusions. The goal was to see if daily bitopertin pills for 8 months could help control the disease by reducing the need for transfusions or increasing hemoglobin levels.
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Contacts and locations
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Locations
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National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States
Conditions
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