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Gene therapies. Beremagene geperpavec (Vyjuvek): treatment of wounds. [ 2] Exagamglogene autotemcel (Casgevy): treatment for sickle cell disease. [ 11] Lovotibeglogene autotemcel (Lyfgenia): treatment for sickle cell disease. [ 11]
DrugBank. DB15572. UNII. S53L777GM8. KEGG. D12749. Exagamglogene autotemcel, sold under the brand name Casgevy, is a gene therapy used for the treatment of sickle cell disease [1][3] and transfusion-dependent beta thalassemia. [1] It was developed by Vertex Pharmaceuticals and CRISPR Therapeutics.
Takeaways from AP's report on access to gene therapies for rare diseases. LAURA UNGAR. June 21, 2024 at 12:42 AM. The promise of gene therapy looms large for families dealing with rare, genetic ...
The Centers for Disease Control and Prevention says 100,000 Americans are living with sickle cell disease. FDA approves first gene therapies to treat sickle cell disease Skip to main content
Xanomeline/trospium, also known under the brand name KarXT, is an investigational oral dual-drug fixed-dose combination of xanomeline and trospium. It is undergoing a phase 3 clinical trial for the treatment of schizophrenia. Xanomeline is a functionally preferring muscarinic M4 and M1 receptor agonist that readily passes into the central ...
The FDA approves the first of two gene therapies this month to treat sickle cell disease. Though expensive and difficult, it promises a better life. FDA approves gene therapy for sickle cell ...
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