Gene Therapy for Duchenne: Translating clinical guidance into meaningful patient understanding скачать в хорошем качестве

Gene Therapy for Duchenne: Translating clinical guidance into meaningful patient understanding

Gene therapy is a major step forward in treating Duchenne muscular dystrophy, offering hope to slow disease progression with a single infusion. But it’s not simple. Deciding whether—and when—to seek gene therapy involves many factors. In this webinar, Parent Project Muscular Dystrophy (PPMD) and the Muscular Dystrophy Association (MDA) review guidelines for gene therapy in DMD produced in partnership with cardiologists from the Advanced Cardiac Therapies Improving Outcomes Network (ACTION Network) and experts from 15 leading MDA and PPMD clinical care centers. The webinar covers: • Current eligibility criteria for gene therapy in Duchenne muscular dystrophy and how these may apply to their child or loved one. • Key factors in choosing a qualified treatment center, including experience with gene therapy, multidisciplinary care, and long-term follow-up capabilities. • How to prepare for gene therapy treatment, including medical evaluations, insurance navigation, and emotional readiness for families. • Recognizing potential side effects of gene therapy and understand strategies for monitoring and managing them safely. • What to expect after treatment, including follow-up care, monitoring outcomes, and ongoing support and resources for families.