Natural history studies are vital for understanding disease progression, particularly in rare diseases. They provide a baseline to evaluate the effectiveness of new treatments. Our latest whitepaper explores the benefits and challenges associated with these studies.

Benefits of natural history studies

These studies are instrumental in identifying disease variability and subtypes, which is crucial for personalised medicine. They also influence the design of clinical trials, enhancing their effectiveness and relevance.

Impact and integration of genetic testing

In conditions like Duchenne Muscular Dystrophy and Cystic Fibrosis, natural history studies, enriched with genetic testing, have significantly advanced our understanding. This integration allows for the development of targeted treatments tailored to the genetic profiles of individual diseases, greatly improving treatment outcomes.

Navigating challenges

The whitepaper addresses the ethical and logistical challenges of conducting these studies, such as patient recruitment and data collection. It emphasises how FDA guidance helps navigate these complexities, ensuring the studies are both scientifically robust and ethically sound.

For a detailed exploration of how natural history studies are reshaping drug development, download our whitepaper.



natural history studies whitepaper

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