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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]
This was the second gene therapy treatment to be approved in Europe. [241] In October, Chinese scientists reported they had started a trial to genetically modify T cells from 10 adult patients with lung cancer and reinject the modified T cells back into their bodies to attack the cancer cells.
Exagamglogene autotemcel is the first cell-based gene therapy treatment utilizing CRISPR/Cas9 gene editing technology to be approved by the US Food and Drug Administration (FDA). [13] The most common side effects include low levels of platelets and white blood cells, mouth sores, nausea, musculoskeletal pain, abdominal pain, vomiting, febrile ...
By comparison, research earlier this year showed medical expenses for current sickle cell treatments, from birth to age 65, add up to about $1.6 million for women and $1.7 million for men.
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 ...
Treatment of people aged twelve years of age and older with solid tumors that: have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion, are locally advanced or metastatic or where surgical resection is likely to result in severe morbidity, and have progressed following treatment or have no satisfactory alternative therapy [2] Resmetirom
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