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

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

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

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

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

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