selected publications
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Low toxicity and favorable overall survival in relapsed/refractory B-ALL following CAR T cells and CD34-selected T-cell depleted allogeneic hematopoietic cell transplant.
Bone marrow transplantation.
2020
Academic Article
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Times cited: 10 -
The clinical severity of hemoglobin S/Black (A γδβ)0 -thalassemia.
2017
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Times cited: 4 -
Addressing challenges of clinical trials in acute pain: The Pain Management of Vaso-occlusive Crisis in Children and Young Adults with Sickle Cell Disease Study.
Clinical trials (London, England).
2016
Academic Article
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Times cited: 15 -
Silent cerebral infarcts in very young children with sickle cell anaemia are associated with a higher risk of stroke.
British journal of haematology.
2015
Academic Article
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Times cited: 35 -
Long-term safety and efficacy of factor IX gene therapy in hemophilia B.
The New England journal of medicine.
2014
Academic Article
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Times cited: 911 - Developments in the treatment of hemophilia B: focus on emerging gene therapy. The application of clinical genetics. 2013 Review GET IT
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Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant.
Blood.
2013
Academic Article
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Times cited: 207 -
Severe pandemic H1N1 and seasonal influenza in children and young adults with sickle cell disease.
Blood.
2010
Academic Article
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Times cited: 63