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Publication
The potential price and access implications of the cost-utility and budget impact methodologies applied by NICE in England and ICER in the US for a novel gene therapy in Parkinson's disease
Journal of market access & health policy -
Publication
Addressing the value of gene therapy and enhancing patient access to transformative treatments
Molecular Therapy -
Publication
Gene therapy of hemoglobinopathies: progress and future challenges
Human Molecular Genetics -
Publication
Regeneration X: The payer perspective on gene therapy
British Journal of Healthcare Management -
Grant Abstract
Consumer Perspectives on the Promise of CF Gene Therapy
In this study, the Education Development Center, Inc., in collaboration with the International Association of Cystic Fibrosis Adults (IACFA), Temple University, St. Vincent's Hospital, and…- Attitude
- behavioral /social science research tag
- Belief
- Clinical Research
- Cystic Fibrosis
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Video
ELSIcon2022 Paper: The 'Lived Experience' of Pediatric Gene Therapy - A Scoping Review
ELSIcon2022 • Paper • May 27, 2022 Laura Kimberly, Cara Hunt, Ryan Dieudonne, Alix Hall, Pat Furlong, Andrew McFadyen, Alison Bateman-House, Jennifer DeSante-Bertkau Little is known about patients…- Motivation to participate in research
- Gene therapy and gene editing
- Paper
- Pre-Recorded
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News
Ethical Issues in Pediatric Gene Therapy Clinical Trials
The Working Group on Pediatric Gene Therapy and Medical Ethics (PGTME) was created in 2019 to study ethical issues surrounding research in genetic interventions for pediatric populations. The group…- pediatric gene therapy
- Pediatric Research
- Informed Consent
- Research Ethics
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Publication
Gene therapy for neuromuscular disorders: Prospects and ethics
Archives of Disease in Childhood -
Publication
The ethics of gene therapy for hemophilia: A narrative review
Journal of Thrombosis and Haemostasis