Beginning the Journey - diagnosed 0-3 years

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Gene Therapy and Gene Editing Symposium

A brief overview of the strategy guiding efforts in gene therapy and gene editing, as well as critical updates from the companies in this space. Moderated discussion included.

Jeffrey Chamberlain, PhD

Moderators

Reed Clark, PhD

Chief Science Officer, Ultragenyx Gene Therapy

Ultragenyx

Olivier Danos, PhD

Chief Scientific Officer

REGENXBIO

Jennifer Hodge, PhD

Team Lead, Rare Neurology USMA

Pfizer

Alison Mcvie-Wylie, PhD

Vice President, Disease Area Executive, DMD

Vertex Pharmaceutical

Carl Morris, PhD

Chief Scientific Officer

Solid Biosciences

Deanna Tucker, PharmD

Senior Medical Science Liaison

Sarepta Therapeutics

Inclusión creativa

Una presentación interactiva enfocada en aprovechar la creatividad en la vida cotidiana con actividades, juegos y ejercicios inclusivos, además de utilizar tecnología/equipos adaptables para superar las expectativas.

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

Involucrarse con CureDuchenne

Una cálida bienvenida del equipo de CureDuchenne y unas palabras de algunas familias campeonas de CureDuchenne sobre cómo lograron el éxito en la recaudación de fondos al unir a su comunidad.

Paul and Laura Heaton

Calves to Cure DMD

Tim and Laura Revell

CureDuchenne Champion Family

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

Next Generation Technology Panel

A brief overview of the rationale behind next generation technology, as well as presentations from the companies in this space. Moderated discussion included.

Erin Brubaker

Chief Operating Officer

Code Bio

Michael Kelly, PhD

Chief Scientific Advisor

CureDuchenne

Joe McIntosh, MD

Chief Medical Officer

Gennao Bio

Genine Winslow, MSc

CEO & President

Chameleon Biosciences