RPS19 deficient Diamond-Blackfan anemia

Clinical trials for RPS19 deficient Diamond-Blackfan anemia explained in plain language.

Never miss a new study

Get alerted when new RPS19 deficient Diamond-Blackfan anemia trials appear

Sign up with your email to follow new studies for RPS19 deficient Diamond-Blackfan anemia, keep track of the ones that matter, and come back to a personal dashboard instead of checking manually.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

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…

Matched conditions: RPS19 DEFICIENT DIAMOND-BLACKFAN ANEMIA

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

Last updated Jul 23, 2026 00:00 UTC

RPS19 deficient Diamond-Blackfan anemia

Get alerted when new RPS19 deficient Diamond-Blackfan anemia trials appear

Gene therapy could free kids from lifelong blood transfusions