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Study of stem cell transplant effects in patients with sickle cell disease

Condition: Blood Disorders / Sickle Cell Disease
Investigator: Monica Bhatia, MD
Status: Closed
This study is being done to see if giving your child a lower dose of chemotherapy (drug therapy), than has been used historically in bone marrow transplant, to kill his/her own diseased cells before he/she receives a bone marrow transplant, will possibly result in the permanent control of your child's sickle…
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Study of EDIT-301 in Patients with Severe Sickle Cell Disease (SCD)

Condition: Blood Disorders / Sickle Cell Disease
Investigator: Monica Bhatia, MD
Status: Closed
The purpose of this study is to find out if the study medicine called EDIT-301 will help in the treatment of Severe Sickle Cell Disease (SCD) and how safe it is to be used in people. EDIT-301 (study medicine) is a new investigational therapy which uses the patient's own stem…
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A study for subjects with Lupus nephritis using an oral study drug MLN9708

Condition: Autoimmune Disorders / Lupus
Investigator: Anca Askanase, MD, MPH
Status: Closed
The purpose of this study is to evaluate the safety and tolerability of an experimental investigational drug called ixazomib citrate (MLN9708) in patient with Lupus Nephritis. Lupus nephritis is a condition when the kidney is affected in the autoimmune disease known as systemic lupus erythematosus (SLE). SLE is a disease in…
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