NIH Commits Millions in Grant Funding for Rare Disease Research

According to a story from, the National Institutes of Health (NIH) has doled a total of $31 million on grants this year to 20 different research teams. The goal? To further rare disease research and develop new therapies. There are as many as 7,000 rare diseases known to science, but only about five percent of them actually have FDA approved treatments.

The groups of researchers committed to rare diseases that have been awarded grants are dubbed the Rare Diseases Clinical Research Network (RDCRN). The goal of grouping all of the teams together in this consortia is to foster a more collaborative research method that encourages scientists to share important data with each other that can help us understand how rare diseases can progress and can accelerate the development of new treatments and diagnostic approaches.

While a rare disease may affect anywhere from a handful to tens of thousands of people, when combined together there are at least 25 million people in the US that are directly affected by them. Children especially are affected to a disproportionate degree. However, the fact that a single rare disease may affect a very limited number of individuals can make them particularly difficult to study effectively. Research funding can also be challenging to obtain.

The RDCRN first appeared as a result of the 2002 Rare Diseases Act. While the group is primary focused on the US, with over 350 sites of research in the country to date, there have also been more than 50 sites in 22 countries total. Studies conducted by the RDCRN have encompassed more than 56,000 participants.

This year, the RDCRN has added a total of five new consortia into the fold. Each of these new groups has a specific research focus and are situated in different regions of the country. Some of the diseases being studies by the new groups include myasthenia gravis, leukodystrophy, congenital disorders of glycosylation, hyperphenylalaninemia disorders, and congenital and perinatal infections.

Many of the latest grants have been focused on projects related to the preparedness to conduct clinical trials. This means that collaboration is critical and prior research such as natural history studies and biomarker research in order to measure the impact of treatment needs to be completed. The support of the NIH plays a critical role in improving rare disease research.

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