Rare disease advocates, because of their diseases’ small population of patients, can find it challenging to capture the interest of researchers and drug developers to invest the money, time, and energy that is needed to discover and develop a therapy. One solution is to drive collaborations to leverage the limited resources of foundations and researchers and advance their efforts to a point where drug company may be willing to take over development. Support of Accelerated Research for Niemann-Pick C, or SOAR-NPC, represents such a model. We spoke to Dr. Cristin Davidson, a researcher and project manager for SOAR-NPC, about the organization’s approach, how its tackled various preclinical challenges common to rare diseases, and why the success it has enjoyed should be replicated by others.
RARECast is a weekly series by Daniel S. Levine. Levine is an award-winning business journalist who has reported on the life sciences, economic development, and business policy issues throughout his 25-year career. He founded Levine Media Group in 2013, which produces The Bio Report and RARECast podcasts. Read his full bio here.