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FDA awards over $38m for advancement of rare disease treatments

The grants and contracts, which were funded by the FDA’s Orphan Products Grants Program, will support clinical trials, natural history studies and regulatory science tools related to rare diseases.

More than $25m, spread over the next four years, was awarded to 11 clinical trials, seven of which are studies of rare cancers, mostly targeting cancers of the brain and peripheral nerves.

An additional eight grants totalling over $11m were awarded to natural history studies, which look closely at how specific diseases progress over time.

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