inherited anemias

Trials

45 participants · First posted Jan 12, 2026

Recruiting

NCT07649213

Congenital Hemolytic and Dyserythropoietic Anemias

400 participants · First posted Jun 16, 2026

Recruiting

NCT07459816

Genomic of CONgenital Sideroblastic Anemias

20 participants · First posted Mar 10, 2026

Drugs

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inherited macular degeneration program

ABCA4 gene therapy (AAV, inherited retinal dystrophy)

Small Molecule

Approved

Deferoxamine(Desferal)

Manufacturer: App pharmaceuticals llc, Bedford laboratories div ben venue laboratories inc +3 more

Conditions: Aluminum overload, Chronic Iron Overload +2 more

C₂₅H₄₈N₆O₈

Organizations

View all 7 →

The Center For Comprehensive Care and Diagnosis of Inherited Blood Disorders

United States

ProQR Therapeutics

Avalo Therapeutics

United States

News

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last year

mRNA Delivery to CD34+ Cells via Lipid Nanoparticles Shows Promise in Rhesus Monkeys

last year

Fulcrum's Pociredir SCD Trial Advances with Key Data Readouts Expected in 2025

last year

Trials

Luspatercept in Patients Affected With Rare Inherited Anemias

Congenital Hemolytic and Dyserythropoietic Anemias

Genomic of CONgenital Sideroblastic Anemias

Drugs

inherited macular degeneration program

ABCA4 gene therapy (AAV, inherited retinal dystrophy)

Deferoxamine(Desferal)

Organizations

The Center For Comprehensive Care and Diagnosis of Inherited Blood Disorders

ProQR Therapeutics

Avalo Therapeutics

News

mRNA Delivery to CD34+ Cells via Lipid Nanoparticles Shows Promise in Rhesus Monkeys

Fulcrum's Pociredir SCD Trial Advances with Key Data Readouts Expected in 2025

Agios Pharmaceuticals Advances Tebapivat for Sickle Cell Disease and Hemolytic Anemias