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Start Over Collections Health Policy and Services Research ✖Remove constraint Collections: Health Policy and Services Research Subjects Genetic Therapy ✖Remove constraint Subjects: Genetic Therapy

1. 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

3. 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

5. 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

6. 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

7. 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.

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

Publication:
Silver Spring, MD : Center for Biologics Evaluation and Research, January 2020
Subject(s):
Drug Contamination
Genetic Therapy
Genetic Vectors
Retroviridae
Virus Replication
United States
United States. Department of Health and Human Services.
United States. Food and Drug Administration.