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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]
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 ...
The first medication approved to treat seizures associated with CDD is ganaxolone (Ztalmy), approved by the FDA in March 2022 [25] and by the EMA in July 2023. [26] Other efforts to develop small molecule therapeutics for CDD include phase 2 and phase 3 trials already underway or completed, and others in earlier stages of development.
The FDA approved Phase I clinical trials on thalassemia major patients in the US for 10 participants in July. [201] The study was expected to continue until 2015. [191] In July 2012, the European Medicines Agency recommended approval of a gene
(Reuters) -The U.S. health regulator has approved Vertex Pharmaceuticals and CRISPR Therapeutics' gene therapy to treat a rare blood disorder requiring regular blood transfusions, in patients 12 ...
(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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