Congenital, Hereditary, and Neonatal Diseases and Abnormalities: News and Research on Genetic Diseases, Inborn

MeSH tree: C16.320

Medications

FDA approves Waskyra for Wiskott-Aldrich syndrome

The U.S. Food and Drug Administration has approved Waskyra (etuvetidigene autotemcel) as the first cell-based gene therapy for the treatment of Wiskott-Aldrich syndrome (WAS).

Diseases, Conditions, Syndromes

Iron overload prevalent in sickle cell disease, study finds

Iron overload is prevalent among adults with sickle cell disease (SCD), according to a study presented at the annual meeting of the American Society of Hematology, held from Dec. 6 to 9 in Orlando, Florida.

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