The mission of the Working Group on Paediatric Gene Therapy and Medical Ethics (PGTME) is to advance research, policy, and education regarding ethical issues surrounding paediatric gene therapy trials. They seek to promote improved understanding of challenges and nascent best practices for ethical research across the evolving landscape of genetic technologies.
The group is part of NYU Langone’s Division of Medical Ethics. The group formed in 2019 to identify and address emerging ethical issues surrounding investigational gene-based therapies in paediatric populations including issues related to research design, patient autonomy, sibling and family involvement, fairness in access, and the quality of information patients and families receive.
Their YouTube channel hosts webinars that may be of interest to the DMD community including (but not limited to):
Trial on Hold: Balancing Expectations and Understanding of Clinical Investigations