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Strimvelis: treatment for adenosine deaminase deficiency (ADA-SCID) Talimogene laherparepvec (Imlygic): treatment for melanoma in patients who have recurring skin lesions [17] Tisagenlecleucel (Kymriah): treatment for B cell lymphoblastic leukemia [18] Valoctocogene roxaparvovec (Roctavian): treatment for hemophilia A [19] [20] [21]
Voretigene neparvovec is an approved gene therapy to treat Leber's hereditary optic neuropathy. [94]: 1354 alipogene tiparvovec, a treatment for pancreatitis caused by a genetic condition, and Zolgensma for the treatment of spinal muscular atrophy both use an adeno-associated virus vector. [78]: 2647
Pages in category "Approved gene therapies" The following 25 pages are in this category, out of 25 total. This list may not reflect recent changes. A.
The Vertex-CRISPR therapy has a U.S. list price of $2.2 million, while bluebird's is $3.1 million. Both therapies, pitched as one-time treatments, will be available in early 2024.
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 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 ...
Approved gene therapies (25 P) C. CAR T-cell therapy (7 P) ... List of gene therapies; 0–9. 2002 French gene therapy trials; A. Advanced Therapy Medicinal Product;
Last year, Britain approved a gene therapy for a fatal genetic disorder that had a list price of £2.8 million ($3.5 million). England's National Health Service negotiated a significant ...