Diamond-Blackfan anemia 1
MONDO:0007110Any 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
8 clinical trials for this condition and its sub-types.
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Gene therapy could free kids from lifelong blood transfusions
Disease control Recruiting nowThis 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: PHASE1 • Sponsor: Apriligen, Inc. • Aim: Disease control
Last updated Jul 23, 2026 00:00 UTC
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New anemia drug enters early human testing
Symptom relief Recruiting nowThis early-stage study tests a new drug called APG-5918 in healthy volunteers and people with anemia (low red blood cells). The main goal is to check safety and how the body handles the drug, while also seeing if it can raise hemoglobin levels. About 105 participants will take pa…
Phase: PHASE1 • Sponsor: Ascentage Pharma Group Inc. • Aim: Symptom relief
Last updated Jun 27, 2026 12:08 UTC
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Hospital tests if its own anemia rules actually get followed
Knowledge-focused Recruiting nowThis study looks at whether doctors at Siriraj Hospital follow a new guideline for treating anemia in patients before major surgery. Anemia is common in surgical patients and can lead to more blood transfusions and worse outcomes. The researchers will track 93 anemic patients to …
Sponsor: Siriraj Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:11 UTC
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Anemia type may predict surgery danger
Knowledge-focused Recruiting nowThis study is observing 1000 adults having moderate- to high-risk surgery to see if the type and severity of anemia they have before surgery affects their risk of complications like heart injury, kidney injury, or stroke. Researchers will compare patients with iron-deficiency ane…
Sponsor: Peking Union Medical College Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:00 UTC