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Displaying 1 - 2 of 2
Condition: Neurological Disorders / Cerebral Palsy
Investigator: Jason Carmel, MD, PhD
Status: Currently Recruiting
The Weinberg Family Cerebral Palsy Center's (WFCPC) research team maintains a cerebral palsy (CP) patient registry of more than 1,000 enrolled patients. This registry contains information collected from children and adults with CP. The goal of the registry is to help us learn more about CP and neuromuscular diseases, to understand the quality of life…
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 cells, modifies them by CRISPR technology, and infuses them back to the…