RPS19 DEFICIENT DIAMOND-BLACKFAN ANEMIA

Clinical trials for RPS19 DEFICIENT DIAMOND-BLACKFAN ANEMIA explained in plain language.

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Trials to join now! 1

Gene therapy may free kids with rare anemia from lifelong blood transfusions

Disease control Recruiting now

This early-stage trial tests a gene therapy called APR-2020 in children and teens with a specific type of Diamond-Blackfan anemia (DBA). The treatment uses the patient's own blood stem cells, modified to correct the genetic defect, and given as a one-time infusion. The goal is to…

Matched conditions: RPS19 DEFICIENT DIAMOND-BLACKFAN ANEMIA

Phase: PHASE1 • Sponsor: Apriligen, Inc. • Aim: Disease control

Last updated May 01, 2026 15:57 UTC

RPS19 DEFICIENT DIAMOND-BLACKFAN ANEMIA

Get alerted when new RPS19 DEFICIENT DIAMOND-BLACKFAN ANEMIA trials appear

Gene therapy may free kids with rare anemia from lifelong blood transfusions