5th Gene Therapy for Muscular Disorders

  • 2025
  • Boston, MA, United States


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.

5th Gene Therapy for Muscular Disorders covers topics such as:

  • 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

5th Gene Therapy for Muscular Disorders might be held in Boston, United States in 2025.


  • Boston, Massachusetts, United States

More Details

2599-5396 US Dollar (Estimated)
Hanson Wade

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Please, check "Gene Therapy for Muscular Disorders" official website for possible changes, before making any traveling arrangements

Event Categories

Health & Medicine: Genetics, Immunology, Medical technology, Orthopedics, Pharma
Technology: Biotechnology

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