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Considerations for Administering Hemoglobinopathy Gene Therapies From a Pharmacist’s Perspective
20 Apr 2024 12:52 GMT
… 2023, the first 2 gene therapy products, Vertex Pharmaceuticals… today, the topic was gene therapy for hemoglobinopathies and particularly … regimen, or of course, prior to the therapy infusion itself … be held, resumed, or possibly switched throughout the …
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Identification and Verification of Ferroptosis-Related Genes in Keratoconus Using Bioinformatics Analysis
20 Apr 2024 12:31 GMT
… , different hypotheses, involving genetics, epigenetics, environmental factors, … hormones, and even pollution or peculiar behavioral traits, … of keratoconus via bioinformatics analysis. This study … the optimal gene biomarkers.
Single-Gene Gene Set Enrichment …
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ACE Gene Mutations (rs577350502) in Early-Onset and Recurrent Myocardial Infarction: A Case Report and Review
20 Apr 2024 12:31 GMT
… years. Following interventional therapy, genetic testing was … or spasm, involves the angiotensin-converting enzyme encoded by the ACE gene … . The ACE gene, encoding angiotensin … Angiotensin converting enzyme gene polymorphism is associated with …
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Do P&T Committees Need Help With Cell and Gene Therapy? | AMCP 2024
20 Apr 2024 02:20 GMT
Expensive cell and gene therapies are fast … stakes intricacies of cell and gene therapies.
Jenny Craven, … getting letters of agreement or single-case agreements in … (delandistrogene moxeparvovec), the first gene therapy for Duchene muscular dystrophy, 43 …
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Cell & Gene Therapies: FDA Regulatory Considerations In 2024
20 Apr 2024 07:42 GMT
… to pause a freshly proposed or ongoing clinical investigation because of … of submitting the essential analyses or documentation or even amending the protocol … -biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products.
See …
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Parkinson Gene Therapy AB-1005 Continues to Show Promising Results at 18 Months
19 Apr 2024 17:20 GMT
… assessing AskBio’s investigational gene therapy AB-1005 in patients … serotype 2 (AAV2) gene therapy containing the human glial … (AEs) associated with the gene therapy or contrast agent. Of note, … -1005 (AAV2-GDNF) gene therapy for multiple system atrophy- …
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Q&A: Cell and gene therapy value assessments need a rework to allow access
19 Apr 2024 17:03 GMT
… factors affecting cell and gene therapy access. ARM is an … cell and gene therapy access?
Paolo Morgese [PM]: Gene therapies have … a new gene therapy is is effective or better than … sustainable.
Cell & Gene Therapy coverage on Pharmaceutical Technology is …
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Cohen Children’s Medical Center Performs First Gene Therapy Treatment For Thalassemia in NYS
19 Apr 2024 16:22 GMT
… as a one-time gene therapy custom designed to treat … in NYS to offer gene therapy treatment for our young … beta-globin gene that results in significantly reduced or absent hemoglobin … steps involved in the Zynteglo gene therapy treatment. In the first …
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Can Clinical Research Spectrum Make The Most of Gene Therapy
19 Apr 2024 16:08 GMT
… the potential long-term consequences. Gene therapy interventions also happen to carry … terms of gene therapy products that are aimed at treating serious or even … to prevent false starts or missed milestones within gene therapy development, specifically as …
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Ocugen Announces Dosing Completion of Subjects with Geographic Atrophy in Cohort 2 of Phase 1/2 ArMaDa Clinical Trial of OCU410—A Modifier Gene Therapy
19 Apr 2024 11:13 GMT
… (AAV-hRORA)—a modifier gene therapy candidate being developed for … “could,” “might,” “will,” “should,” or other words that convey uncertainty … of future events or outcomes to identify these … in ongoing clinical trials or through further analyses of …