Rare Disease

High-Velocity Development: Gene Therapy Vs. Small Molecule

Posted in: Rare Disease, Thought Leadership

Gene therapy development operates at a high velocity compared to small molecule […]

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Rare Disease Clinical Endpoints: Ingenuity Meets Practicality

Posted in: Clinical Operations, Rare Disease, Thought Leadership

Discover why the science that brought you to this point can only […]

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Rare Disease Clinical Endpoints: Ingenuity Meets Practicality

Posted in: Neurodegenerative, Rare Disease, Thought Leadership

Discover why the science that brought you to this point can only […]

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Natural History Studies in Gene Therapy Trials: Benefits, Timing and Execution

Posted in: Clinical Operations, Gene Therapy, Rare Disease, Thought Leadership

Natural history studies (NHS) in gene therapy trials are essential for understanding […]

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