Addressing the Challenges of Rare Disease Drug Development

March 9, 2018

Developing therapeutics for rare diseases can be challenging, and not just because of the small patient populations. Often the progression of a disease is not well understood, the patient population can be heterogenous, and the development of objective and meaningful endpoints for a trial can be difficult. We spoke to Ralf Rosskamp, chief medical officer of Dicerna, about how his company has navigated the various challenges in its effort to develop a treatment for a rare liver disorders, how it finds patients, and how it determines endpoints.

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