Resource Library Archive - Page 6 of 14 - CureDuchenne

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Pilares del viaje de transición

Esta sesión está destinada a familias/cuidadores de adolescentes y adultos con Duchenne. Cubre cuatro pilares del viaje transicional de Duchenne: Continuidad de la atención, Planificación del estilo de vida, Éxito financiero y Escuela/Carrera/Pasatiempos, y es seguido por una sesión de discusión/Preguntas y respuestas.

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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Kent Williams, MD

Pediatric Gastroenterologist

Nationwide Children's Hospital

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Jennifer Shumsky, BSN, RN, CHCO, PACS

Director, Payer Relations

Little Hercules Foundation

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Hawken Miller

Content Strategist

CureDuchenne

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éxito en la escuela

Una presentación interactiva enfocada en definir y construir su equipo en la escuela, abordando los matices de un IEP o plan 504 para una persona con Duchenne, y un ejercicio de juego de roles sobre cómo comunicar de manera efectiva las necesidades de su familia en una reunión escolar.

Doug Levine, PT

CureDuchenne Certified Physical Therapist

CureDuchenne

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Jennifer Ramirez

Executive Director of Special Programs

Belton ISD

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Sheryl Marrazzo

Duchenne Mother, Grandmother and Advocate

4 Jake's Sake | CureDuchenne

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Letter for School Physical Limitations

A letter from Jennifer Wallace Valdes, PT to share with your child’s teachers. This document serves to familiarize them with Duchenne, the physical limitations posed, and reasonable accommodations for the school environment.

Jennifer Wallace Valdes, PT

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REGENXBIO

RGX-202: REGENXBIO’s investigational gene therapy for the treatment of Duchenne Muscular Dystrophy presented by: Johannaz (Naz) Dastgir, DO, Senior Director, Clinical Development Lead at REGENXBIO

A program update on RGX-202, REGENXBIO’s investigational gene therapy program for the treatment of Duchenne.

Contact: Duchenne@regenxbio.com or patientadvocacy@regenxbio.com

Johannaz Dastgir, DO

Senior Director, Clinical Development Lead

REGENXBIO

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Edgewise Therapeutics

Edgewise Therapeutics Industry Presentation

Edgewise Therapeutics is a clinical-stage biopharmaceutical company focused on creating novel precision medicines for the treatment of rare muscle disorders. Our intimate knowledge of integrated muscle physiology at a whole-body level allows us to develop innovative solutions for patients with muscle disease where significant unmet medical need exists. By protecting and improving muscle health, our goal is to dramatically enhance the lives of people living with progressive muscle disorders.

Joanne Donovan, MD, PhD

Chief Medical Officer

Edgewise Therapeutics

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CureDuchenne 2022 Priorities: Research for a Cure and Care for our Community

Paul, Debra and Hawken Miller discuss FUTURES 2022, current advancements in therapies and next generation investments as well as programs CureDuchenne offers to improve the quality of life for those impacted by Duchenne.

Debra Miller and Paul Miller

Founders

CureDuchenne

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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

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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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Exon Skipping and Read Through Agents Panel

A brief introduction on the rationale behind mechanisms of endogenous dystrophin restoration, like exon skipping and read through agent therapies, followed by critical information and updates from the companies in this space. Moderated discussion included.

Barry Byrne, MD, PhD

Professor and Associate Chair

University of Florida, Department of Pediatrics

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Jay Chauhan, PharmD

Medical Science Liaison

NS Pharma

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Brett Crawford, PhD

Executive Director and Head of the Musculoskeletal Therapeutic Area

BioMarin Pharmaceutical Inc.

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Ash Dugar, PhD

Senior Vice President, Medical Affairs

Dyne Therapeutics

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Xiao Shelley Hu, PhD, MS

VP

Wave Life Sciences

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

Chief Operating Officer

PTC Therapeutics

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Nerissa Kreher, MD, MBA

Chief Medical Officer

Entrada Therapeutics

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James McArthur, PhD

President and Chief Executive Officer

PepGen

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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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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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