This resource offers information for parents, caregivers and individuals with Duchenne about the specialized aspects of emergency care for those with Duchenne.
Esta presentación se centra en las pautas nutricionales para las personas con distrofia muscular de Duchenne.
Dietista Nutricionista Registrada
Children's Hospital of Los Angeles
Registered Dietitian Nutritionist
Children’s Hospital of Los Angeles
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
Esta presentación se enfoca en fisioterapia, estiramientos y transferencias seguras para personas con Duchenne y sus cuidadores.
CureDuchenne Fisioterapeuta Certificado
Children's Hospital of Phoenix
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.
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.
CureDuchenne Certified Physical Therapist
CureDuchenne Physical Therapy Program
Occupational Therapist III
Children’s Health and Rehabilitation Services
Quantum Product & Technical Education Manager
Pride Mobility
Esta presentación proporciona información sobre genética básica, salud del portador genético y planificación familiar.
Consejera Genética Certificada
Hospital de Niños de San Antonio
