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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]
In data cited by the FDA ahead of an advisory committee meeting on the therapy in October, 29 of 30 patients reached the trial’s main goal: freedom from a pain crisis for at least 12 months ...
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 ...
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.
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 ...
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