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‘This is not a science problem anymore': Paths emerge for scaling up rare disease medicine

In 2014, the National Institutes of Health posted an estimate that at the current pace of drug development and approval, it would take 1,000 years to get treatments for the 7,000 known rare diseases.


A paper at the time suggested speeding up that work by grouping together similar diseases, and then treating these patients with the same therapy in a single clinical trial.


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Disclaimer: This document is the work product of the N=1 Collaborative (the “N1C”), and is offered as an example only. The N1C is not providing legal or regulatory advice for N=1 trials. This document should not be construed as legal or regulatory advice for any particular purpose. Any future use of this document should be tailored to specific trials based, for example, on pre-clinical data ASO chemistry and mechanism, patient disease, and evolving body of knowledge, and where appropriate, should separately be subjected to legal review.

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