As gene therapy continues to advance through clinical trials, many new concepts, terms and issues arise, including those related to research design, patient autonomy, sibling and family involvement, fairness in access and the quality of information patients and families receive.

As part of PPMD’s Gene Therapy Initiative, PPMD has been supporting NYU’s Division of Medical Ethics who in partnership with PPMD formed the Pediatric Gene Therapy and Medical Ethics (PGTME) Working Group. The goal of the working group is to advance research, policy, and education, by promoting improved understanding of the challenges and best practices for ethical research across the evolving landscape of genetic therapies. This working group includes a range of stakeholders to include patient advocacy, law, medicine, clinical research, and the biopharmaceutical industry.

PGTME’S EDUCATIONAL LECTURE SERIES

Last year, the PGTME Working Group hosted a lecture series on a range of topics relevant to our community, including the FDA perspective, AAV serotypes, acquired and innate immunity, informed consent and the patient perspective.

PGTME is bringing back this popular series, building on the success and useful information that came out of the original series. It is our hope that these lectures will provide the community with educational tools as we move through development of potential gene therapies for Duchenne. All families are welcome to sign up to attend any or all of the week-long sessions:

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Topics Include:

November 29th: Pediatric Gene Therapy Research in the Context of Uncertain Risk-Benefit

• Moderator: Katherine Beaverson, Senior Director, Patient Advocacy Lead, Pfizer
• Adam Hartman, Program Director, Division of Clinical Research, NINDS/NIH
• Ryan Fischer, Chief Advocacy Officer, Parent Project Muscular Dystrophy
• Cláudia Hirawat, Executive Chair, VOZ Advisors
• Safiyya Gassman, Director, Policy & Public Affairs (Rare Disease & Gene Therapy), Pfizer

November 30th: Trust & Transparency for Trial Participants and Families

• Moderator: Lesha Shah, Assistant Professor of Psychiatry, Mount Sinai Icahn School of Medicine; PGTME Co-Chair
• Heather Lau, Executive Director, Global Clinical Development, Ultragenyx
• Valder Arruda, Researcher, Division of Hematology, The Children’s Hospital of Philadelphia
• Andrew McFadyen, Executive Director, The Isaac Foundation
• Aisha Langford, Assistant Professor, Department of Population Health, NYU Grossman School of Medicine

December 1st: The Many Facets of Hope

• Moderator: Moshe Cohn, Clinical Ethicist, Pediatric Intensivist and Palliative Care Physician, Faculty Affiliate, Division of Medical Ethics, NYU School of Medicine
• Jennifer Siedman, Director of Family Engagement, Courageous Parents Network
• Laura Palmer, Chaplain, Cancer Center and Center for Fetal Diagnosis and Treatment, Children’s Hospital of Philadelphia, specializing in palliative care
• Tartania Brown, Clinical Assistant Professor, Department of Family and Social Medicine, Albert Einstein College of Medicine
• Rafael Escandon, Clinical Research Consultant & former SVP, Med Affairs, Policy & Patient Engagement, BridgeBio

December 2nd: The Lived Experience: Social, Emotional, and Practical Aspects

• Moderator: Patrick Moeschen, Coordinator, Adult Advocacy & Engagement, Parent Project Muscular Dystrophy (PPMD); Music Teacher
• Erin Ward, President, MTM-CNM Family Connection; Myotubular Myopathy Mom and Advocate
• Cecilia Valrie, Director, Health Psychology Doctoral Program, Virginia Commonwealth University
• Diana Bharucha-Goebel, Assistant Professor, Children’s National Hospital; Clinical Research Fellow, National Institutes of Health
• Gretchen Egner, English teacher, PPMD Connect Coordinator, Wisconsin

December 3rd: Accountability and Collaboration with Patient Communities

• Moderator: Alison Bateman-House, Assistant Professor, Department of Population Health, NYU Grossman School of Medicine; PGTME Co-Chair
• Liza Marie Johnson, Associate Member, St. Jude Faculty; Program Director, Oncology Hospitalist Medicine; Bioethics Consultant (Clinical & Research Consultations)
• Pat Furlong, President and CEO, Parent Project Muscular Dystrophy
• Lakshmanan Krishnamurti, Chief, Yale Pediatric Hematology and Oncology

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