Growth and Transformation - diagnosed 3-6 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

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Emil Kakkis, PhD

CEO & President

Ultragenyx Pharmaceutical

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Alison Mcvie-Wylie, PhD

Vice President, Disease Area Executive, DMD

Vertex Pharmaceutical

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Dan Levy, MD

VP and Development Head, Rare Neurology Pfizer

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Carsten Bönnemann, MD

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

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Kevin Flanigan, MD

Director, Center for Gene Therapy Nationwide Children's Hospital

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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

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Jahannaz Dastgir, DO

Clinical Development Lead REGENXBIO

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Patrick Gonzalez, PhD

Senior Director of Clinical Science Solid Biosciences

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Teji Singh, MD

Vice President, Head of Clinical Development Sarepta Therapeutics

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Dan Levy, MD

VP and Development Head, Rare Neurology Pfizer

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2023 FUTURES: New Science Sessions

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

Dave Anderson, PhD

Chief Scientific Officer CodeBio

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Ranjan Batra, PhD

Senior Vice President, Research & Development Locanabio

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Stan Froehner, PhD

Founder and Chairman Myosana Therapeutics

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Peixin Zhu, PhD

VP of R&D Tevard Biosciences

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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

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Robert Halter, PharmD

Field Medical Director, South/Southwest Santhera Pharmaceuticals

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Karyn Koladicz, MD

Executive Medical Director for Medical Affairs, Neurology PTC Therapeutics

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Linda Marbán , PhD

President, CEO and Director Capricor Therapeutics

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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

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Adam Gold, PhD

Medical Science Liasion

NS Pharma

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Kevin Flanigan, MD

Director, Center for Gene Therapy Nationwide Children's Hospital

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Karin Lucas, Ph.D

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

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Christian Werner, MD

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

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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

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Nerissa Kreher, MD, MBA

Chief Medical Officer

Entrada Therapeutics

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Jane Larkindale, DPhil

VP of Clinical Science PepGen

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Dania Porco, MS, MBA, CGC

Director in Clinical Science BioMarin Pharmaceutical Inc.

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Michael Tillinger, MD

VP of Clinical Development Wave Life Sciences

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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

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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

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Diana Castro, MD

Associate Professor of Pediatrics, Neurology and Neurotherapeutics

University of Texas Southwestern

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Richard Parad, MD, MPH

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

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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

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2024 FUTURES – Action is Therapy

Action is Therapy: Getting Involved With CureDuchenne Why I…fundraise, advocate, attend events, volunteer, engage with CureDuchenne. An overview of CureDuchenne Programs and the champions who are working to Cure Duchenne.

Paul and Laura Heaton

Calves to Cure DMD

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Sheryl Marrazzo

Duchenne Mother, Grandmother and Advocate

4 Jake's Sake | CureDuchenne

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