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Start Over Subjects Genetic Therapy ✖Remove constraint Subjects: Genetic Therapy

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

Publication:
[Boston, Massachusetts] : Institute for Clinical and Economic Review, February 14, 2018
Subject(s):
Genetic Therapy
Leber Congenital Amaurosis -- genetics
Leber Congenital Amaurosis -- therapy
Retinal Diseases -- genetics
Retinal Diseases -- therapy
Retinal Pigment Epithelium -- abnormalities
Mutation
Randomized Controlled Trials as Topic
Treatment Outcome
Visual Acuity
Humans
United States

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

Author(s):
Beaudoin, Francesca L., author
Richardson, Marina, author
Synnott, Patricia G., author
Lancaster, Victoria, author
Fluetsch, Noemi, author
Herce-Hagiwara, Belén, author
Campbell, Jon D., author
Pearson, Steven D., author
Rind, David M., author
Publication:
[Boston, Massachusetts] : Institute for Clinical and Economic Review, July 19, 2022
Subject(s):
beta-Thalassemia -- therapy
Biological Products -- therapeutic use
Genetic Therapy
United States

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

Author(s):
Rind, David M., author
Walton, Surrey M., author
Agboola, Foluso, author
Herron-Smith, Serina, author
Quach, Danny, author
Chapman, Rick M., author
Pearson, Steven D., author
Bradt, Pamela, author
Publication:
[Boston, Massachusetts] : Institute for Clinical and Economic Review, November 20, 2020
Subject(s):
Antibodies, Monoclonal
Genetic Therapy
Hemophilia A -- drug therapy
United States

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

Author(s):
Ellis, Alexandra, author
Mickle, Kristin, author
Herron-Smith, Serina, author
Kumar, Varun M., author
Cianciolo, Laura, author
Seidner, Matt, author
Rind, David, author
Pearson, Steven, author
Thokala, P. (Praveen), author
Stevenson, M. (Matt), author
Publication:
[Boston, Massachusetts] : Institute for Clinical and Economic Review, May 24, 2019
Subject(s):
Genetic Therapy
Muscular Atrophy, Spinal -- drug therapy
United States

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

Publication:
Silver Spring, MD : Center for Biologics Evaluation and Research, January 2020
Subject(s):
Chemistry, Pharmaceutical
Genetic Therapy
Investigational New Drug Application
Drugs, Investigational
Quality Control
United States
United States. Department of Health and Human Services.
United States. Food and Drug Administration.