Advances in the understanding of the biology underlying rare diseases, as well as progress with therapeutic platforms such as nucleic-acid agents, have enabled increased research and development (R&D) on drugs for rare diseases.
Governmental incentives and regulatory initiatives have played a key role too, such as the Orphan Drug Act of 1983 in the USA.
Enjoying our latest content?
Log in or create an account to continue
- Access the most recent journalism from Nature's award-winning team
- Explore the latest features & opinion covering groundbreaking research
or
Acknowledgements
The authors gratefully acknowledge X. Wang and S. Ji from the Center for Drug Evaluation, National Medical Products Administration in China, as well as Z. Yan, Y. Li, C. Ge and J. An from the Tsinghua Clinical Research Institute, Tsinghua University, for their contribution to the work.
Competing Interests
S. Zhou is an employee of Citeline. The other authors declare no competing interests.
