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PGTME Lunchtime Lecture Series: Trust & Transparency for Trial Participants and Families

VIRTUAL - Pediatric Gene Therapy And Medical Ethics (PGTME), is bringing forth a new and fun learning series for all stakeholders within pediatric research and healthcare called the 2021 Annual PGTME Lunchtime Lecture Series. This free series is open to all and will share information on many important topics within gene therapy trials.

If you are not already familiar, the mission of the working group on Pediatric Gene Therapy and Medical Ethics is to advance research, policy, and education regarding ethical issues surrounding gene therapy trials. PGTME seeks to promote improved understanding of challenges and nascent best practices for ethical research across the evolving landscape of genetic technologies.

About this event

PGTME will host multistakeholder discussions on five different ethical topics in pediatric gene therapy research:

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

  • Cecelia 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, Director, Parent Project Muscular Dystrophy

  • Lakshmanan Krishnamurti, Chief, Yale Pediatric Hematology and Oncology

To log in and watch each of the series, please use this zoom link: For more information, please visit:

If you are interested in joining iCAN to learn more about the pediatric research and healthcare community, please visit

Don't forget, November 30th marks Giving Tuesday.. please support iCAN and share how important it is to support the voice of youth members and families. #GivingTuesday #iCANMakeADifference #iCAN #iCANResearch #iCANGivingTuesday

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