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Gene therapy approaches to replace a faulty gene with a healthy gene have been proposed and are being studied for treating some genetic diseases. As of 2017, 11.1% of gene therapy clinical trials targeted monogenic diseases.
Exagamglogene autotemcel (Casgevy): treatment for sickle cell disease. [11] Gendicine: treatment for head and neck squamous cell carcinoma; Idecabtagene vicleucel (Abecma): treatment for multiple myeloma [12] Lovotibeglogene autotemcel (Lyfgenia): treatment for sickle cell disease. [11] Nadofaragene firadenovec (Adstiladrin): treatment for ...
Beta thalassemia is a heritable disorder, characterized by the inability to make beta globin protein, and in turn reduced functioning of hemoglobin (which beta globin is a part of). [10] In December 2023, the European Medicines Agency recommended approval for a cell based gene therapy that works through the CRISPR/Cas9 system.
The Food and Drug Administration on Friday approved Pfizer’s treatment for a rare genetic bleeding disorder, making it the company’s first-ever gene therapy to win clearance in the U.S.
(Reuters) -The U.S. Food and Drug Administration approved Pfizer's gene therapy for hemophilia B on Friday, the second such therapy for the rare bleeding disorder that typically requires regular ...
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