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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 ...
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]
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]
Xanomeline/trospium. 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 ...
August 25, 2024 at 2:00 AM. After a lull of nearly 2 decades, the Food and Drug Administration (FDA) has approved some novel drugs for the treatment of Alzheimer’s disease since 2021. Most of ...
The gene therapy will compete with Australia-based CSL Behring’s Hemgenix, a similar treatment that won FDA approval for hemophilia B in 2022. That drug has a similar list price of $3.5 million ...
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