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Beta-Thalassemia

Prevalence: ~5M

hemoglobin

Official·OMIM:613985 Official·ORPHA848

Inherited anemia caused by insufficient production of the beta chain of hemoglobin.

Approved Therapies

Casgevy (exagamglogene autotemcel)

FDA Approved1/16/2024CRISPR-Cas9

Associated Genes

HBBHemoglobin Subunit Beta

Chromosome 11Autosomal Recessive