by: Parent Project Muscular Dystrophy
We are excited to announce the launch of
PPMD’s Gene Therapy Hub!
Gene therapy has made significant strides in Duchenne, with one therapy approved and other potential therapies in various stages of development. As our community continues to see progress, many new concepts and terms arise, ones that families have not had to consider before.
PPMD’s Gene Therapy Hub aims to empower and inform families like yours, providing insights and guidance to help you better understand the dynamic landscape of gene therapy.
We’re here to support you on this journey, offering clarity and knowledge as we navigate this evolving field together.
PPMD’s Gene Therapy ResourcesGene Therapy 101
Get an overview of the basics of gene therapy in Duchenne with a 3-minute video and a glossary of gene therapy terms to know.
Research Insights
Stay informed about the latest developments in the field of gene therapy for Duchenne and learn about eligibility and access to existing therapies.
Navigating Clinical Trials
Learn more about the clinical trials for gene therapies, including trials that are currently enrolling or expected to begin enrollment, and how PPMD can help you better understand the different gene therapies and various trials.
PPMD Video Series: Exploring Topics in Gene Therapy
In our new video series, PPMD’s team walks you through specific topics in gene therapy such as understanding adeno-associated virus (AAV) in gene therapy and gene replacement with micro-dystrophin.
VIDEO SERIES 1: UNDERSTANDING AAV IN GENE THERAPY
Researchers in Duchenne are currently using an adeno-associated virus (AAV) to deliver the genetic code for dystrophin or other proteins into muscle cells in the body. Learn about the properties of AAV and why it is used, immune challenges, as well as potential safety concerns.
VIDEO SERIES 2: GENE REPLACEMENT WITH MICRO-DYSTROPHIN
Hear from the team about why gene replacement with a micro-dystrophin is the most prevalent approach to treating Duchenne with gene therapy, as well as some of the challenges regarding its limitations and immune response to transgene.
