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Titles
- Animal research will make gene therapy happen1
- Betibeglogene autotemcel for beta thalassemia: effectiveness and value : final evidence report1
- Chemistry, manufacturing, and control (CMC) information for human gene therapy investigational new drug applications (INDs)1
- Gene therapies for sickle cell disease: final evidence report1
- Gene therapy for hemophilia B and an update on gene therapy for hemophilia A: effectiveness and value : final evidence report1
- Human gene therapy1
- Human gene therapy for hemophilia1
- Human gene therapy for rare diseases1
- Human gene therapy for retinal disorders1
- Interpreting sameness of gene therapy products under the orphan drug regulations1
- Letter from Bernard D. Davis to Maxine Singer1
- S12 nonclinical biodistribution considerations for gene therapy products: guidance for industry1
- Spinraza and Zolgensma for spinal muscular atrophy: effectiveness and value : final evidence report1
- Studying multiple versions of a cellular or gene therapy product in an early-phase clinical trial: guidance for industry1
- Testing of retroviral vector-based human gene therapy products for replication competent retrovirus during product manufacture and patient follow-up1
- The National Institutes of Health presents a conference on human gene therapy: December 4-6, 1991, Masur Auditorium, National Institutes of Health, Bethesda, Maryland1
- Valoctocogene roxaparvovec and emicizumab for hemophilia A without inhibitors: effectiveness and value : final report1
- Voretigene neparvovec for biallelic RPE65-mediated retinal disease: effectiveness and value : final evidence report1