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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]
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 ...
Choroideremia is an inherited genetic eye disease with no approved treatment, leading to loss of sight. In March researchers reported that 12 HIV patients had been treated since 2009 in a trial with a genetically engineered virus with a rare mutation (CCR5 deficiency) known to protect against HIV with promising results. [221] [222]
The US Food and Drug Administration on Friday approved two gene-based treatments for sickle cell disease, including the first therapy that uses the gene-editing technique CRISPR, opening a new era ...
Regulators on Friday approved two gene therapies for sickle cell disease that doctors hope can cure the painful, inherited blood disorder that afflicts mostly Black people in the U.S. The Food and ...
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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