Innovative Collaboration Model Drives Treatment for Rare Disease

Rare disease advocates, because of their diseases’ small population of patients, can find it challenging to muster the interest of researchers and drug developers to invest the money, time, and energy needed to discover and develop a therapeutic. 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 Cristin Davidson, a researchers 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.