5th Gene Therapy for Muscular Disorders is a conference dedicated to genetic therapies targeting muscular disorders, including Limb-Girdle Muscular Dystrophy (LGMD), Duchenne Muscular Dystrophy (DMD), X-Linked Myotubular Myopathy and Pompe Disease.
Topics
- Muscle Gene Therapy: How can we Effectively & Safely Express Transgenes in the Largest Body Compartment
- Translating from Preclinical to Clinical in Duchenne’s
- Clinically Meaningful Endpoints & Outcome Measures for Muscular Disorders
- Optimizing Gene Therapy Delivery with Next Generation Capsids & Muscle Specific Promoters
- Exploring CRISPR-based Gene Editing for Muscular Disorders
- Quality & Quantity: Addressing Manufacturing Challenges for Large Muscular Disorder Doses
- Preclinical Model Challenges to Effectively Determine Dose
- Lessons Learned: Mitigating Toxicity & Immunogenicity Challenges
- Clinical Challenges for Muscular Disorders
