FANCONI ANEMIA COMPLEMENTATION GROUP A

Clinical trials for FANCONI ANEMIA COMPLEMENTATION GROUP A explained in plain language.

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Doctors test One-Time gene fix for rare blood disorder in kids

Disease control Ongoing

This trial is testing a one-time gene therapy for children with a rare, serious genetic disorder called Fanconi anemia type A. Doctors take a patient's own blood stem cells, fix the faulty gene in a lab, and then return the corrected cells to the patient. The goal is to prevent t…

Matched conditions: FANCONI ANEMIA COMPLEMENTATION GROUP A

Phase: PHASE2 • Sponsor: Rocket Pharmaceuticals Inc. • Aim: Disease control

Last updated Mar 30, 2026 14:29 UTC

FANCONI ANEMIA COMPLEMENTATION GROUP A

Get alerted when new FANCONI ANEMIA COMPLEMENTATION GROUP A trials appear

Doctors test One-Time gene fix for rare blood disorder in kids