Spreading Wisdom - diagnosed 6+ years

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2023 FUTURES: Friday Keynote – On the Cutting Edge of Research

The science and research-focused day of FUTURES will begin with an engaging keynote panel featuring key opinion leaders who will discuss the successes, learnings, challenges, and future of this disease space.

Michael Kelly, PhD

Chief Scientific Advisor

CureDuchenne

Emil Kakkis, PhD

CEO & President

Ultragenyx Pharmaceutical

Alison Mcvie-Wylie, PhD

Vice President, Disease Area Executive, DMD

Vertex Pharmaceutical

Dan Levy, MD

VP and Development Head, Rare Neurology Pfizer

Carsten Bönnemann, MD

Chief, Neuromuscular and Neurogenetic Disorders of Childhood Section National Institute of Neurological Disorders and Stroke/NIH

Kevin Flanigan, MD

Director, Center for Gene Therapy Nationwide Children's Hospital

2023 FUTURES: Gene Therapy and Gene Editing Sessions

A brief primer on the gene therapy and gene editing approach to treating Duchenne, and updates from relevant companies in the space. These presentations will be followed by a moderated Q&A opportunity.

Alison Mcvie-Wylie, PhD

Vice President, Disease Area Executive, DMD

Vertex Pharmaceutical

Jahannaz Dastgir, DO

Clinical Development Lead REGENXBIO

Patrick Gonzalez, PhD

Senior Director of Clinical Science Solid Biosciences

Teji Singh, MD

Vice President, Head of Clinical Development Sarepta Therapeutics

Dan Levy, MD

VP and Development Head, Rare Neurology Pfizer

2023 FUTURES: New Science Sessions

A session that highlights novel and pre-clinical research in Duchenne.

Dave Anderson, PhD

Chief Scientific Officer CodeBio

Ranjan Batra, PhD

Senior Vice President, Research & Development Locanabio

Stan Froehner, PhD

Founder and Chairman Myosana Therapeutics

Peixin Zhu, PhD

VP of R&D Tevard Biosciences

2023 FUTURES: Preserving Muscle Sessions

A brief primer on the muscle preservation and anti-inflammatory treatment approach to Duchenne, and updates from relevant companies in the space. These presentations will be followed by a moderated Q&A opportunity.

Abby Bronson

Vice President, Patient Advocacy & External Innovation

Edgewise Therapeutics

Robert Halter, PharmD

Field Medical Director, South/Southwest Santhera Pharmaceuticals

Karyn Koladicz, MD

Executive Medical Director for Medical Affairs, Neurology PTC Therapeutics

Linda Marbán , PhD

President, CEO and Director Capricor Therapeutics

2023 FUTURES: RNA targeted Therapeutics Sessions Part 1

A brief primer on the RNA-targetd therapeutic approach to treating Duchenne, and updates from relevant companies in the space. These presentations will be followed by a moderated Q&A opportunity.

Ash Dugar, PhD

Senior Vice President, Medical Affairs

Dyne Therapeutics

Adam Gold, PhD

Medical Science Liasion

NS Pharma

Kevin Flanigan, MD

Director, Center for Gene Therapy Nationwide Children's Hospital

Karin Lucas, Ph.D

West Medical Science Liaison Team Lead, Global Medical Affairs Sarepta Therapeutics

Christian Werner, MD

Executive Director, Global Medical Affairs, and Global Duchenne Muscular Dystrophy Lead PTC Therapeutics

2023 FUTURES: RNA targeted Therapeutics Sessions Part 2

Continued updates from relevant companies in the space. These presentations will be followed by a moderated Q&A opportunity.

Mark Stahl, MD, PhD

Senior Medical Director, Clinical Development

Avidity Biosciences

Nerissa Kreher, MD, MBA

Chief Medical Officer

Entrada Therapeutics

Jane Larkindale, DPhil

VP of Clinical Science PepGen

Dania Porco, MS, MBA, CGC

Director in Clinical Science BioMarin Pharmaceutical Inc.

Michael Tillinger, MD

VP of Clinical Development Wave Life Sciences

2023 FUTURES: Trailblazers The Future of Care Panel

A panel discussion featuring leading healthcare providers that are making a positive impact on variety of aspects of care for Duchenne.

Natalie Truba, PhD

Clinical Psychologist

Nationwide Children's Hospital

Pradeep P.A. Mammen, MD, FACC, FAHA

Associate Professor of Medicine and Co-Director

Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, Director for Translational Research for the Advanced Heart Failure and Transplant Cardiology Program, UT Southwestern Medical Center

Diana Castro, MD

Associate Professor of Pediatrics, Neurology and Neurotherapeutics

University of Texas Southwestern

Richard Parad, MD, MPH

Associate Professor of Pediatrics / Department of Pediatric Newborn Medicine Harvard Medical School Department of Pediatrics / Brigham and Women's Hospital

2023 FUTURES: Welcome & Saturday Keynote Address

Justin & Patrick are back! From wheelchair races in the courtyard to personally connecting with families, parents, caregivers and individuals with Duchenne, their experience with the community last year was hugely impactful on them. The duo is back to share new stories from their last accessible journey through Camino de Santiago and encourage others the find and foster purpose – no matter the circumstances.

Justin Skeesuck and Patrick Gray

Inspirational Keynote Speakers

I'll Push You

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