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

Kevin Eggan, PhD

Group Vice President, Head of Research and Early Development

BioMarin

Leslie Magnus, MD

Vice President, Medical Affairs

NS Pharma

James McArthur, PhD

President and Chief Executive Officer

PepGen

Lianna Orlando, PhD

Senior Director of Research

CureDuchenne

Mark Stahl, MD, PhD

Senior Medical Director, Clinical Development

Avidity Biosciences

Deanna Tucker

Senior Medical Science Liaison

Sarepta Therapeutics

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

Olivier Danos, PhD

Chief Scientific Officer

REGENXBIO

Emil Kakkis, PhD

CEO & President

Ultragenyx Pharmaceutical

Michael Kelly, PhD

Chief Scientific Advisor

CureDuchenne

Alison Mcvie-Wylie, PhD

Vice President, Disease Area Executive, DMD

Vertex Pharmaceutical

Carl Morris, PhD

Chief Scientific Officer

Solid Biosciences

Eric Olson, PhD

Chief Scientific Advisor

Vertex Pharmaceuticals

Matthew Pletcher, PhD

Division Head of Gene Therapy Research & Technical Operations

Astellas Gene Therapies

Deanna Tucker

Senior Medical Science Liaison

Sarepta Therapeutics

Genine Winslow, MSc

CEO & President

Chameleon Biosciences

2021 FUTURES: Keynote Panel

An inspiring conversation about living in the moment with a rare diagnosis.

Sean Baumstark

Co-Founder

Two Disabled Dudes

Kyle Bryant

Co-Founder

Two Disabled Dudes

Jake Marrazzo

Living with Duchenne

Glenn Olesen

Living with Duchenne

GlennDane Adapt and Conquer

Tanner Rico

Living with Duchenne

A Family Guide to Dealing With Duchenne Muscular Dystrophy

This resource presents the history of Duchenne muscular dystrophy and provides a basic understanding of the genetics, management and treatment of the diesease.

Samiya Ahmad, MD

Assistant Professor of Pediatrics, Child Neurology and Sleep Medicine | Medical Director, Comprehensive Neuromuscular Center

Baylor College of Medicine | Children's Hospital of San Antonio

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

Ana Christensen, MPH

Lead Project Manager, Strategic Solutions

TRiNDS

Leslie Porter

Family, Duchenne Advocate

Family, Duchenne Advocate

Michael Kelly, PhD

Chief Scientific Advisor

CureDuchenne

Michaela Walker, MPH

Project Manager

University of Kansas Medical Center