I have the same mission as Benioff Children’s Hospital Oakland: to treat and improve the outcome for every child and family who comes here for care and support.
Where I see patients (2)
Selected research
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Betibeglogene autotemcel gene therapy in patients with transfusion-dependent, severe genotype β-thalassaemia (HGB-212): a non-randomised, multicentre, single-arm, open-label, single-dose, phase 3 trial.
Lancet (London, England)
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Hematopoietic Cell Transplant compared with Standard Care in Adolescents and Young Adults with Sickle Cell Disease.
Blood advances
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Zinc finger nuclease-mediated gene editing in hematopoietic stem cells results in reactivation of fetal hemoglobin in sickle cell disease.
Scientific reports
Clinical trials
Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoi...
The adverse event rate will be summarized using descriptive statistics, together with 95% confidence intervals where appropriate. No formal statistical hypothesis testing will be performed. Adverse events defined: failure of engra...
Recruiting
More about this studyA Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell Disease (GRASP, BMT CTN 2001)
Each patient will be classified as either a success or a failure (binary endpoint). Success is defined as a complete absence of severe VOEs (defining VOE as a painful event or ACS with no medically determined cause other than a va...
Recruiting
More about this studyContact me
Translation, please