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Inherited Hematological Diseases
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Inherited Hematological Diseases trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which Inherited Hematological Diseases trials you may qualify forThis is a phase 3, prospective, open-label, single-arm, single-dose, multicenter study investigating the efficacy, safety, and tolerability of CSL222 (AAV5-hFIX…
The goal of this clinical trial is to evaluate the impact of SCThrive (a behavioral self-management intervention) on patient activation, self-management behavio…
Background: Fanconi anemia (FA) is a rare, inherited cancer syndrome. FA causes a range of physical issues. Children with FA may have abnormal features; these…
The purpose of this study is to evaluate the efficacy and safety of Coagadex in treating active bleeds and to manage peri-operative bleeding in participants wit…
The purpose of this study is to evaluate the effectiveness of nipocalimab compared with placebo in reducing the risk of severe fetal and neonatal alloimmune thr…
The purpose of this study is to assess the efficacy and safety of nipocalimab in reducing the risk of severe fetal and neonatal alloimmune thrombocytopenia (FNA…
This is an observational study of children with mild, moderate, or severe hemophilia A who are receiving damoctocog alfa pegol, and are between 7 to \<12 years…
Background: Clinical Genetics Branch (CGB) researchers study individuals and populations at high genetic risk of cancer in order to improve our understanding o…
To evaluate the outcome of a prednisolone and low dose methotrexate based protocol in Down syndrome children with ALL (DS-ALL) in an Asia-wide study. The treatm…