NLM Digital Collections

Search

Search Constraints

Start Over You searched for: Subjects Genetic Therapy Remove constraint Subjects: Genetic Therapy

Search Results

3. Voretigene neparvovec for biallelic RPE65-mediated retinal disease: effectiveness and value : final evidence report

5. Betibeglogene autotemcel for beta thalassemia: effectiveness and value : final evidence report

7. Valoctocogene roxaparvovec and emicizumab for hemophilia A without inhibitors: effectiveness and value : final report

8. Spinraza and Zolgensma for spinal muscular atrophy: effectiveness and value : final evidence report

9. Chemistry, manufacturing, and control (CMC) information for human gene therapy investigational new drug applications (INDs)

11. Testing of retroviral vector-based human gene therapy products for replication competent retrovirus during product manufacture and patient follow-up

13. Human gene therapy for hemophilia

15. Gene therapies for sickle cell disease: final evidence report

16. Gene therapy for hemophilia B and an update on gene therapy for hemophilia A: effectiveness and value : final evidence report

18. Human gene therapy