Research and Clinical Trials

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The Research and Clinical Trials section provides information on topics related to the Duchenne research pipeline, varying treatment approaches, navigating clinical trials and more.

2021 FUTURES: Exon Skipping Panel

Information and updates on exon skipping therapies and research from experts in the Duchenne community.

Ash Dugar, PhD

Senior Vice President, Medical Affairs

Dyne Therapeutics

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Kevin Eggan, PhD

Group Vice President, Head of Research and Early Development

BioMarin

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Leslie Magnus, MD

Vice President, Medical Affairs

NS Pharma

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James McArthur, PhD

President and Chief Executive Officer

PepGen

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Lianna Orlando, PhD

Senior Director of Research

CureDuchenne

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Mark Stahl, MD, PhD

Senior Medical Director, Clinical Development

Avidity Biosciences

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Deanna Tucker, PharmD

Senior Medical Science Liaison

Sarepta Therapeutics

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2021 FUTURES: Gene Therapy & Gene Editing Symposium

Information and updates on the gene therapy and gene editing landscape from experts in the field.

**We experienced a technical issue during this session. Please reach out with questions to Futures@CureDuchenne.org if needed.

Beth Belluscio, MD, PhD

Global Clinical Lead, Rare Neurological Disorders

Pfizer

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Olivier Danos, PhD

Chief Scientific Officer

REGENXBIO

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Emil Kakkis, PhD

CEO & President

Ultragenyx Pharmaceutical

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Michael Kelly, PhD

Chief Scientific Advisor

CureDuchenne

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Alison Mcvie-Wylie, PhD

Vice President, Disease Area Executive, DMD

Vertex Pharmaceutical

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Carl Morris, PhD

Chief Scientific Officer

Solid Biosciences

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Eric Olson, PhD

Chief Scientific Advisor

Vertex Pharmaceuticals

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Matthew Pletcher, PhD

Division Head of Gene Therapy Research & Technical Operations

Astellas Gene Therapies

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Deanna Tucker, PharmD

Senior Medical Science Liaison

Sarepta Therapeutics

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Genine Winslow, MSc

CEO & President

Chameleon Biosciences

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A Holistic View of Clinical Trials

This panel discussion provides a robust conversation about how clinical trials intersect with family life in the Duchenne community.

Tiffany Cook, MS, CCC-SLP

Senior Director

CureDuchenne Cares

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Ana Christensen, MPH

Lead Project Manager, Strategic Solutions

TRiNDS

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Leslie Porter

Family, Duchenne Advocate

Family, Duchenne Advocate

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Michael Kelly, PhD

Chief Scientific Advisor

CureDuchenne

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Michaela Walker, MPH

Project Manager

University of Kansas Medical Center

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DMD Clinical Trials: An Overview

This resource presents information on the clinical trial process, common clinical trial terms, updated trial processes due to telehealth, and more.

Lauren Morgenroth, MS, CGC

Chief Operating Officer

TRiNDS

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Ana Christensen, MPH

Lead Project Manager, Strategic Solutions

TRiNDS

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The Family’s Role in Research Panel

A deep dive into the world of clinical and academic research, including input from multiple stakeholders, with the family at the center of it all.

Matthew Alexander, PhD

Assistant Professor in Pediatric Neurology and Genetics

Children’s of Alabama | University of Alabama at Birmingham

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Diana Castro, MD

Associate Professor of Pediatrics, Neurology and Neurotherapeutics

University of Texas Southwestern

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Tiffany Cook, MS, CCC-SLP

Senior Director

CureDuchenne Cares

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Joanna Kemp, BSN, RN

Senior Director of Clinical Operations | Director, CureDuchenne Link

CureDuchenne

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Lauren Morgenroth, MS, CGC

Chief Operating Officer

TRiNDS

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Clint & Leslie Porter

CureDuchenne Champion Family

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