Diamond-Blackfan anemia 1

MONDO:0007110

Any Diamond-Blackfan anemia in which the cause of the disease is a mutation in the RPS19 gene.

Also known as: DBA, Diamond-Blackfan Anaemia type 1, Diamond-Blackfan Anemia type 1, Diamond-Blackfan anaemia caused by mutation in RPS19, Diamond-Blackfan anemia 1, Diamond-Blackfan anemia caused by mutation in RPS19, RPS19 Diamond-Blackfan anaemia, RPS19 Diamond-Blackfan anemia

10 clinical trials for this condition and its sub-types, 1 tagged with Diamond-Blackfan anemia 1 itself.

Follow this condition to get notified about new trials

Where it sits in the disease tree

Browse by category →

Part of

↑ Diamond-Blackfan anemia (17)

Including sub-types (10) Tagged with Diamond-Blackfan anemia 1 (1)

Trials to join now! 1

Gene therapy could free kids from lifelong blood transfusions

Disease control Recruiting now

This early-stage trial tests a one-time gene therapy called APR-2020 in 4 children with a severe form of Diamond-Blackfan anemia. The treatment uses the child's own blood stem cells, modified to correct the genetic defect, and given back as an infusion. The goal is to see if it i…

Phase 1 • Sponsor: Apriligen, Inc. • Aim: Disease control

Last updated Jul 23, 2026 00:00 UTC

Diamond-Blackfan anemia 1

Where it sits in the disease tree

Gene therapy could free kids from lifelong blood transfusions