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.
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
Senior Director, Clinical Development Lead
REGENXBIO
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.
Sarepta Pipeline Update for the Duchenne Community
Contact Information for Patient Affairs Team: advocacy@sarepta.com
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 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
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 rationale behind next generation technology, as well as presentations from the companies in this space. Moderated discussion included.
