2021 FUTURES Quality of Life Panel: Defining Happiness

Focused on the intersection between physical needs and living a fulfilling life.

Tiffany Cook, MS, CCC-SLP

Senior Director

CureDuchenne Cares

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Doug Levine, PT

CureDuchenne Certified Physical Therapist

CureDuchenne

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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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Tom McNalley, MD

Associate Professor of Pediatrics

University of California San Francisco Benioff's Children's Hospitals

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Brenda Wong, MD

Professor of Pediatrics and Neurology and Director

UMass Duchenne Muscular Dystrophy Program at the University of Massachusetts Medical School

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2021 FUTURES Quality of Life Panel: Cognition in Duchenne

Focused on cognitive abilities and psychological wellness in individuals living with Duchenne.

Tiffany Cook, MS, CCC-SLP

Senior Director

CureDuchenne Cares

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Missy Dixon, MS, PhD

Visiting Assistant Professor, Department of Pediatrics, Divisions of Pediatric Neurology, Psychiatry and Behavioral Health

University of Utah

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Danielle Forrest, OTR, OTD

Occupational Therapist III

Children’s Health and Rehabilitation Services

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Matthew Harmelink, MD

Pediatric Neurologist

Medical College of Wisconsin

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Mathula Thangarajh, MD, PhD, FAAN

Assistant Professor of Neurology

Virginia Commonwealth University

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2021 FUTURES Quality of Life Panel: Making Decisions

Focused on empowering families to make informed decisions about their child’s care / personal care across all junctures of the Duchenne journey.

John Brandsema, MD

Child Neurologist and Neuromuscular Section Head

Children’s Hospital of Philadelphia

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Tiffany Cook, MS, CCC-SLP

Senior Director

CureDuchenne Cares

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Michael Kelly, PhD

Chief Scientific Advisor

CureDuchenne

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Sheryl Marrazzo

Duchenne Mother, Grandmother and Advocate

4 Jake's Sake | CureDuchenne

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Christina Trout, RN, MSN

Advanced Practice Nurse, Neuromuscular Program

Department of Pediatrics, University of Iowa Stead Family Children’s Hospital

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Jennifer Wallace Valdes, PT

CureDuchenne Certified Physical Therapist

CureDuchenne Physical Therapy Program

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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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2021 FUTURES: Keynote Panel

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

Sean Baumstark

Co-Founder

Two Disabled Dudes

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Kyle Bryant

Co-Founder

Two Disabled Dudes

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Jake Marrazzo

Living with Duchenne

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Glenn Olesen

Living with Duchenne

GlennDane Adapt and Conquer

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Tanner Rico

Living with Duchenne

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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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