Gene Therapy
Ethical issues surrounding gene therapy trials and proposed clinical trial endpoints for gene therapy for NF1, NF2, and Schwannomatosis.
Leadership
About This Group
The goals of the gene therapy working group are (1) to discuss ethical issues surrounding trials of gene therapy and (2) to propose clinical trial endpoints for gene therapy that can lead to drug approval for NF1, NF2, and Schwannomatosis.
Current group leader: Scott Plotkin, MD, PhD
The Gene Therapy group was formed in January 2022. Initial projects focus on defining the important ethical issues surrounding gene therapy in the NF/SWN patient population and establishing appropriate, measurable endpoints for future clinical trials.
Additional Information
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Working Group Snapshot & Focus Updates
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Publications & References
Gene therapy for neurofibromatosis: ethical considerations and proposed endpoints for clinical trials
Bakker A, Plotkin SR, et al. Clin Trials. 2024;21(1):88–97.
Documents & Resources
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Join or Contact This Group
Membership is open to all interested parties. Meetings are primarily held by teleconference.
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