Beginning the Journey - diagnosed 0-3 years

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

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Reed Clark, PhD

Chief Science Officer, Ultragenyx Gene Therapy

Ultragenyx

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Olivier Danos, PhD

Chief Scientific Officer

REGENXBIO

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Jennifer Hodge, PhD

Team Lead, Rare Neurology USMA

Pfizer

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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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Deanna Tucker, PharmD

Senior Medical Science Liaison

Sarepta Therapeutics

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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

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Danielle Forrest, OTR, OTD

Occupational Therapist III

Children’s Health and Rehabilitation Services

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Austin Edenfield, ATP

Quantum Product & Technical Education Manager

Pride Mobility

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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

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Tim and Laura Revell

CureDuchenne Champion Family

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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

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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

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Michael Kelly, PhD

Chief Scientific Advisor

CureDuchenne

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Joe McIntosh, MD

Chief Medical Officer

Gennao Bio

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Genine Winslow, MSc

CEO & President

Chameleon Biosciences

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Beginning the Journey - diagnosed 0-3 yearsGrowth and Transformation - diagnosed 3-6 yearsSpreading Wisdom - diagnosed 6+ years