Ferroheme

Generic Name: Ferroheme

Brand Names: -

Drug Type: Small Molecule

Chemical Formula: C_₃₄H_₃₂FeN_₄O_₄

CAS Number: 14875-96-8

Unique Ingredient Identifier: 42VZT0U6YR

Associated Conditions: -

Associated Therapies: -

Clinical Trials

First of Its Kind Gene Therapy Model Offers Hope for X-Linked Sideroblastic Anemia Treatment

Researchers at CHOP and UPenn developed a gene therapy model targeting X-linked sideroblastic anemia (XLSA), a rare anemia caused by ALAS2 gene mutations. This breakthrough, using a lipid nanoparticle platform, could transform treatment for XLSA and other diseases, offering an alternative to current treatments like blood transfusions and stem cell transplants.

Drug Development Updates

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