Enrolling by Invitation
An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID
NCT04049084
- Conditions
- Adenosine Deaminase Deficiency, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA Repair-Deficiency Disorders, +10 more
- Locations
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- Mattel Children's Hospital UCLA/Ronald Reagan UCLA Medical Center Los Angeles, California, United States
- UCL Great Ormond Street Institute of Child Health London, United Kingdom