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 study is testing a one-time gene therapy for children with Fanconi Anemia type A, a rare genetic disorder that causes bone marrow failure. Doctors take the patient's own blood stem cells, correct the faulty gene in a lab, and then return the corrected cells to the patient. T…

Matched conditions: FANCONI ANEMIA COMPLEMENTATION GROUP A

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

Last updated Apr 02, 2026 10:42 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