NLM Digital Collections

Search

Search Constraints

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

Search Results

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

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

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

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

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

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