FUTURES

Clear all

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

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

Comunidad de cuidadores

Una discusión interactiva sobre el viaje único de los cuidadores y herramientas para unir y fortalecer a la comunidad de Duchenne en su experiencia compartida.

Laura McLinn

Founder

Best Day Ever Foundation

Marissa Penrod

Co-Founder

The Duchenne Family Assistance Program

Leslie Porter

Family, Duchenne Advocate

Family, Duchenne Advocate

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

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

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

Jay Chauhan, PharmD

Medical Science Liaison

NS Pharma

Brett Crawford, PhD

Executive Director and Head of the Musculoskeletal Therapeutic Area

BioMarin Pharmaceutical Inc.

Ash Dugar, PhD

Senior Vice President, Medical Affairs

Dyne Therapeutics

Xiao Shelley Hu, PhD, MS

VP

Wave Life Sciences

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

Chief Operating Officer

PTC Therapeutics

Nerissa Kreher, MD, MBA

Chief Medical Officer

Entrada Therapeutics

James McArthur, PhD

President and Chief Executive Officer

PepGen

Mark Stahl, MD, PhD

Senior Medical Director, Clinical Development

Avidity Biosciences

Deanna Tucker, PharmD

Senior Medical Science Liaison

Sarepta Therapeutics

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