FANCONI ANEMIA COMPLEMENTATION GROUP A
Clinical trials for FANCONI ANEMIA COMPLEMENTATION GROUP A explained in plain language.
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Gene therapy for rare blood disease tracked for 15 years
Disease control OngoingThis study follows 9 people with Fanconi Anemia who received a one-time gene therapy to fix a faulty gene. Researchers will monitor their health and blood counts for 15 years to see if the treatment remains safe and effective. No new treatment is given during this follow-up perio…
Matched conditions: FANCONI ANEMIA COMPLEMENTATION GROUP A
Sponsor: Rocket Pharmaceuticals Inc. • Aim: Disease control
Last updated May 22, 2026 13:55 UTC
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Gene therapy offers hope for kids with rare bone marrow disease
Disease control OngoingThis study tests a gene therapy for children with Fanconi anemia subtype A, a rare genetic condition that leads to bone marrow failure. Doctors take the child's own blood stem cells, fix the faulty gene in a lab, and put the corrected cells back into the body. The goal is to rest…
Matched conditions: FANCONI ANEMIA COMPLEMENTATION GROUP A
Phase: PHASE2 • Sponsor: Rocket Pharmaceuticals Inc. • Aim: Disease control
Last updated May 22, 2026 13:51 UTC