A warm welcome from Debra, Paul and Hawken, and an informative presentation on CureDuchenne’s timeline of contributions and investments, and how these inform our future vision.
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.
Vice President, Patient Advocacy & External Innovation
Edgewise Therapeutics
Vice President of Clinical Development
Capricor Therapeutics
An interactive discussion on the unique journey of caregivers, and tools to unite and strengthen the Duchenne community in their shared experience.
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.
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.
CureDuchenne Certified Physical Therapist
CureDuchenne Physical Therapy Program
Occupational Therapist III
Children’s Health and Rehabilitation Services
Quantum Product & Technical Education Manager
Pride Mobility
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.
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.
Professor and Associate Chair
University of Florida, Department of Pediatrics
Executive Director and Head of the Musculoskeletal Therapeutic Area
BioMarin Pharmaceutical Inc.
Senior Medical Director, Clinical Development
Avidity Biosciences
Senior Medical Science Liaison
Sarepta Therapeutics
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.
Chief Science Officer, Ultragenyx Gene Therapy
Ultragenyx
Vice President, Disease Area Executive, DMD
Vertex Pharmaceutical
Senior Medical Science Liaison
Sarepta Therapeutics
Presentations by CureDuchenne Champion families on how they found success in fundraising by bringing their community together.
