Gene therapy in rare diseases faces a unique set of challenges—from navigating complex regulatory environments and Chemistry, Manufacturing, and Controls (CMC) barriers to overcoming small patient populations, trial design limitations, and long-term safety monitoring. Explore how gene therapy is transforming the treatment landscape for rare diseases—and what it takes to bring these breakthrough therapies to life.

Inside This White Paper

This white paper dives into the realities of clinical development for gene therapy in rare neuromuscular diseases, providing strategic insight for sponsors, biotech innovators, and clinical research professionals.

Download now to learn:

• How gene therapy is revolutionizing rare disease care
  Understand why gene therapy is uniquely suited to treat rare diseases at the source—and how it’s reshaping standards of care for neuromuscular and other genetic conditions.
• Strategies to overcome regulatory and operational complexity
  Explore expert guidance on managing CMC hurdles, regulatory variability across regions, and evolving requirements for long-term patient monitoring.
• The role of patient-centered trial design in rare disease success
  Learn how inclusive enrollment criteria, specialized site engagement, and ongoing patient support are essential to trial feasibility and retention.
• Why expert partnerships accelerate success in gene therapy trials
  See how working with a CRO experienced in gene therapy in rare diseases can provide the infrastructure, insight, and cross-functional collaboration needed to deliver results.

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