Paving the way for a cure: researchers test first step for gene therapy in rare blood disease
NCT ID NCT07186179
Summary
This study is a first step to see if future gene therapy might be possible for people with Diamond Blackfan Anemia (DBA), a rare and serious inherited blood disorder. Researchers will give two drugs to 10 participants with DBA to see if they can move enough of their own stem cells from the bone marrow into the bloodstream. The goal is to measure if enough cells could be collected for a future treatment, but no cells will actually be collected or treated in this study.
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Contacts and locations
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Study contacts
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Contact
Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Cohen Children's Medical Center
RECRUITINGNew Hyde Park, New York, 11040, United States
Contact
Contact Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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