Synonyms ANEMIA WITH MULTINUCLEATED ERYTHROBLASTS, ANEMIA, CONGENITAL DYSERYTHROPOIETIC, TYPE I, ANEMIA, CONGENITAL DYSERYTHROPOIETIC, TYPE II + [110] |
Introduction A familial disorder characterized by ANEMIA with multinuclear ERYTHROBLASTS, karyorrhexis, asynchrony of nuclear and cytoplasmic maturation, and various nuclear abnormalities of bone marrow erythrocyte precursors (ERYTHROID PRECURSOR CELLS). Type II is the most common of the 3 types; it is often referred to as HEMPAS, based on the Hereditary Erythroblast Multinuclearity with Positive Acidified Serum test. |
Target |
Mechanism KDM1A inhibitors [+1] |
Active Org. |
Originator Org. |
Active Indication |
Inactive Indication- |
Drug Highest PhasePreclinical |
First Approval Ctry. / Loc.- |
First Approval Date20 Jan 1800 |
Target- |
Mechanism- |
Active Org. |
Originator Org. |
Active Indication |
Inactive Indication- |
Drug Highest PhasePreclinical |
First Approval Ctry. / Loc.- |
First Approval Date20 Jan 1800 |
Target |
Mechanism CDAN1 modulators [+1] |
Active Org. |
Originator Org. |
Active Indication |
Inactive Indication- |
Drug Highest PhasePreclinical |
First Approval Ctry. / Loc.- |
First Approval Date20 Jan 1800 |
Start Date17 Jul 2023 |
Sponsor / Collaborator- |
Start Date15 Mar 2023 |
Sponsor / Collaborator- |
Start Date01 Oct 2022 |
Sponsor / Collaborator- |