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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Beginning the Journey - diagnosed 0-3 yearsGrowth and Transformation - diagnosed 3-6 yearsSpreading Wisdom - diagnosed 6+ years

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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Beginning the Journey - diagnosed 0-3 yearsGrowth and Transformation - diagnosed 3-6 yearsSpreading Wisdom - diagnosed 6+ years

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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Beginning the Journey - diagnosed 0-3 yearsGrowth and Transformation - diagnosed 3-6 yearsSpreading Wisdom - diagnosed 6+ years