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RPS19 Deficient Diamond-Blackfan Anemia
Clinical trial pipeline · Data from ClinicalTrials.gov
See which RPS19 Deficient Diamond-Blackfan Anemia trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which RPS19 Deficient Diamond-Blackfan Anemia trials may be worth asking aboutNorth America
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NOTE: This is a research study and is not meant to be a substitute for clinical genetic testing. Families may never receive results from the study or may receiv…
Background: A prospective cohort of Inherited Bone Marrow Failure Syndrome (IBMFS) will provide new information regarding cancer rates and types in these disor…
The purpose of this study is to collect and store samples and health information for current and future research to learn more about the causes and treatment of…
Brief summary The goal of this clinical trial is to learn if APR-2020 is safe and can help treat Diamond-Blackfan Anemia (DBA) in adolescents and children. The…
This is a data collection study that will examine the general diagnostic and treatment data associated with the reduced-intensity chemotherapy-based regimen pai…
The objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), double…
This study's goal is to determine the frequency and severity of acute graft versus host disease, to evaluate incidence of primary and secondary graft rejection,…
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions. With a simple…