Beginning the Journey - diagnosed 0-3 years

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2019 FUTURES: FAMILY Panel

This panel discusses genetic carrier health, mental health and coping skills, and assistance for Duchenne families.

Virginia Kimonis, MD, MRCP

Professor, Department of Pediatrics, Division of Genetics and Genomic Medicine

University of California, Irvine Medical Center, Children’s Hospital of Orange County

Lauren Morgenroth, MS, CGC

Chief Operating Officer

TRiNDS

Ashley Yee, MSW

Director of Patient Engagement

Global Genes

Laurie Miller, CSW

Clinical Social Worker

Children's Hospital of Los Angeles

Leona Filis

Special Needs Attorney

The Filis Law Firm, P.C.

Kelly Maynard

Co-Founder

The Duchenne Family Assistance Program

Marissa Penrod

Co-Founder

The Duchenne Family Assistance Program

2019 FUTURES: FITNESS Panel

This panel discusses mental and physical fitness in individuals with Duchenne.

Tom McNalley, MD

Associate Professor of Pediatrics

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

Missy Dixon, MS, PhD

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

University of Utah

Jennifer Wallace Valdes, PT

CureDuchenne Certified Physical Therapist

CureDuchenne Physical Therapy Program

Paige Lembeck, PhD

Pediatric Psychologist  

Yale Child Study Center

2019 FUTURES: FUNCTION Panel

This panel discusses clinical care across multiple disciplines for individuals with Duchenne.

Eric Ball, MD

Pediatrician

Southern Orange County Pediatric Associates

Brenda Wong, MD

Professor of Pediatrics and Neurology and Director

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

Andrew Darmahkasih, MD

PGY-2, UC Irvine

Children’s Hospital of Orange County Pediatric Residency Program

John Brandsema, MD

Child Neurologist and Neuromuscular Section Head

Children’s Hospital of Philadelphia

Umme Vahanvaty, MS, RD, CS

Registered Dietitian Nutritionist

Children’s Hospital of Los Angeles

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

Kathryn Wagner, MD, PhD

Director and Professor of Neurology and Neuroscience

Center for Genetic Muscle Disorders at Kennedy Krieger Institute and Johns Hopinks School of Medicine

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

Missy Dixon, MS, PhD

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

University of Utah

Danielle Forrest, OTR, OTD

Occupational Therapist III

Children’s Health and Rehabilitation Services

Matthew Harmelink, MD

Pediatric Neurologist

Medical College of Wisconsin

Mathula Thangarajh, MD, PhD, FAAN

Assistant Professor of Neurology

Virginia Commonwealth University

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

Doug Levine, PT

CureDuchenne Certified Physical Therapist

CureDuchenne

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

Tom McNalley, MD

Associate Professor of Pediatrics

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

Brenda Wong, MD

Professor of Pediatrics and Neurology and Director

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

2021 FUTURES Quality of Life Panel: Easing the Burden

Focused on engaging families in conversations about the load they carry and how to manage life as a caregiver.

Taylor Berhow, MSW, MPA

Social Worker and Neuro-behavioral Health Specialist

University of Utah Health

Tiffany Cook, MS, CCC-SLP

Senior Director

CureDuchenne Cares

Richard Kravitz, MD

Professor of Pediatric Pulmonary and Sleep Medicine

Duke University

Marissa Penrod

Co-Founder

The Duchenne Family Assistance Program

Lori Safford

Duchenne Mother and Advocate

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

Tiffany Cook, MS, CCC-SLP

Senior Director

CureDuchenne Cares

Michael Kelly, PhD

Chief Scientific Advisor

CureDuchenne

Sheryl Marrazzo

Duchenne Mother, Grandmother and Advocate

4 Jake's Sake | CureDuchenne

Christina Trout, RN, MSN

Advanced Practice Nurse, Neuromuscular Program

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

Jennifer Wallace Valdes, PT

CureDuchenne Certified Physical Therapist

CureDuchenne Physical Therapy Program

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