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The U.S. Food and Drug Administration (FDA) has approved Waskyra (etuvetidigene autotemcel), the first cell-based gene therapy for the treatment of Wiskott-Aldrich Syndrome (WAS).
Waskyra is indicated for pediatric patients six months and older and adults with WAS who have a mutation in the WAS gene and for whom hematopoietic stem cell transportation (HSCT) is appropriate and no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available.
The application was granted Orphan Drug, Rare Pediatric Disease and Regenerative Medicine Advanced Therapy designations.
The FDA granted approval of Waskyra to Fondazione Telethon ETS. This is a first approved cell and gene therapy product from a non-product applicant.
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