Beyond Dystrophin Panel

This conference session provides insight and updates from companies whose therapies target critical aspects of the Duchenne disease process that occur alongside reductions in dystrophin.

Abby Bronson

Vice President, Patient Advocacy & External Innovation

Edgewise Therapeutics

Mindy Cameron

Patient Advocacy Lead

Santhera Pharmaceuticals

Matthew Klein, M.D., M.S., FACS

Chief Operating Officer

PTC Therapeutics

Daniel Paulson, MD

Vice President of Clinical Development

Capricor Therapeutics

Han Phan, MD

Director

Rare Disease Research, LLC

Pillars of the Transition Journey

This session is intended for families/caregivers of teens and adults with Duchenne. It covers four pillars of the transitional Duchenne journey: Continuity of Care, GI-related Care, Medical Privacy, and School/Career/Hobbies, and is followed by a discussion/Q&A session.

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

Hawken Miller

Journalist

Washington Post

Jennifer Shumsky, BSN, RN, CHCO, PACS

Director, Payer Relations

Little Hercules Foundation

Kent Williams, MD

Pediatric Gastroenterologist

Nationwide Children's Hospital

Community of Caregivers

An interactive discussion on the unique journey of caregivers, and tools to unite and strengthen the Duchenne community in their shared experience.

Laura McLinn

Founder

Best Day Ever Foundation

Marissa Penrod

Co-Founder

The Duchenne Family Assistance Program

Leslie Porter

Family, Duchenne Advocate

Family, Duchenne Advocate

Success at School

An interactive presentation focused on defining and building your team at school, addressing the nuances of an IEP or 504 plan for an individual with Duchenne, and a role-playing exercise on how to effectively communicate your family’s needs in a school meeting.

Doug Levine, PT

CureDuchenne Certified Physical Therapist

CureDuchenne

Jennifer Ramirez

Executive Director of Special Programs

Belton ISD

Sheryl Marrazzo

Duchenne Mother, Grandmother and Advocate

4 Jake's Sake | CureDuchenne

Pillars of the Early Journey

This session is intended for families/caregivers of children and adolescents with Duchenne. It covers four pillars of the early Duchenne journey: Multidisciplinary Care, Steroids, Nutrition, and Involvement in Clinical Trials, and is followed by a discussion/Q&A session.

Barry Byrne, MD, PhD

Professor and Associate Chair

University of Florida, Department of Pediatrics

Diana Castro, MD

Associate Professor of Pediatrics, Neurology and Neurotherapeutics

University of Texas Southwestern

Kindann Fawcett, PhD

Post Doctoral Fellow

University of Arkansas for Medical Sciences

Creative Inclusivity

An interactive presentation focused on harnessing creativity in day-to-day life with inclusive activities, games and exercises, as well as utilizing adaptive technology/equipment to surpass expectations.

Jennifer Wallace Valdes, PT

CureDuchenne Certified Physical Therapist

CureDuchenne Physical Therapy Program

Danielle Forrest, OTR, OTD

Occupational Therapist III

Children’s Health and Rehabilitation Services

Austin Edenfield, ATP

Quantum Product & Technical Education Manager

Pride Mobility

Linking the Community to a Cure

CureDuchenne Link is a centralized data hub which combines clinical data, biosamples, and patient reported data for individuals with Duchenne and Becker muscular dystrophy, as well as carriers. Information and samples provided by participants are coded and available for use by approved researchers across the world.

Ana Christensen, MPH

Lead Project Manager, Strategic Solutions

TRiNDS

Conectando familias con la investigacion

El enfoque del seminario web de hoy es ayudar a educar y conectar a las familias con el panorama de los ensayos clínicos.

Amaris Sánchez-Larragoity, PsyD

Psicóloga licenciada

Diana Castro, MD

Associate Professor of Pediatrics, Neurology and Neurotherapeutics

University of Texas Southwestern