Congenital dyserythropoietic anemia type 1

MONDO:0020337

Congenital dyserythropoietic anemiatype I (CDA I) is a hematologic disorder of erythropoiesis characterized by moderate to severe macrocytic anemia occasionally associated with limb or nail deformities and scoliosis.

Also known as: CDA I, CDA type 1, CDA type I, congenital dyserythropoietic anemia type 1, anemia, dyserythropoietic, congenital type 1, dyserythropoietic anemia, congenital type 1, type I congenital dyserythropoietic anaemia, type I congenital dyserythropoietic anemia

10 clinical trials for this condition and its sub-types, 1 tagged with Congenital dyserythropoietic anemia type 1 itself.

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↑ Congenital anemia (58) ↑ Congenital dyserythropoietic anemia (5)

Sub-types of Congenital dyserythropoietic anemia type 1

Anemia, congenital dyserythropoietic, type 1a 0 trials
Congenital dyserythropoietic anemia type type 1B 0 trials

Including sub-types (10) Tagged with Congenital dyserythropoietic anemia type 1 (1)

Not yet recruiting 1

New hope for rare anemias: drug may cut transfusions

Disease control Not yet recruiting

This Phase 2 trial tests the drug luspatercept (Reblozyl) in 45 people with rare inherited anemias that affect red blood cells. The goal is to see if it can reduce the need for blood transfusions in those who depend on them, or raise hemoglobin levels in those who don't. Particip…

Phase 2 • Sponsor: EuroBloodNet Association • Aim: Disease control

Last updated Jun 27, 2026 08:10 UTC

Congenital dyserythropoietic anemia type 1

Where it sits in the disease tree

New hope for rare anemias: drug may cut transfusions