About Rare Disease Research 

At our center, we conduct clinical trials for patients with rare diseases, whom otherwise would not have the opportunity to receive any treatment or therapy. There are currently nearly 7000 rare diseases and less than 400 treatments approved by the FDA. With the advent of molecular understanding and discovery, we now have a unique opportunity to identify potential treatment through clinical research.  

Our multidisciplinary team is well equipped with years of clinical research experience to carry clinical trials in rare diseases.  Our mission is to remove barriers to investigational therapies for all patients with rare diseases.  

To see a list of all studies we are currently participating in, please visit ClinicalTrials.gov

Our collaborators include

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