This resource presents information on genetic carrier health, carrier testing options and resources, family planning education, and carrier burden information.
Certified Genetic Counselor
Children's Hospital of San Antonio, Baylor College of Medicine
This resource presents the innerworkings of the DMD gene, Duchenne’s impact on the body, and provides and in-depth view of the pipeline of current treatments for Duchenne.
This resource offers information for Emergency Medical Services professionals about the specialized aspects of emergency care for those with Duchenne.
This resource provides multiple handouts to help inform your child’s educator of the specialized needs in a school setting.
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.
This resource presents information on emergency care guidelines for individuals with Duchenne, and tips on how to communicate with emergency care providers in the event of an urgent hospital visit.
Pediatric Critical Care Fellow
Riley Hospital for Children at IU Health
This resource offers information for parents, caregivers and individuals with Duchenne about the specialized aspects of emergency care for those with 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
FibroGen Industry Presentation
FibroGen accelerates the delivery of innovative treatments that operate at the cutting edge of cancer research. Our teams have identified and are developing potential first-in-class medicines for the treatment of life-threatening conditions such as metastatic pancreatic cancer, locally advanced pancreatic cancer (LAPC), and chemotherapy-induced anemia (CIA). We are keenly focused on leveraging the body’s natural pathways to treat cancer and its related conditions.
Executive Medical Director, Clinical Development (Fibrosis/Oncology)
FibroGen
