Driving New Therapy Development for Batten Disease

Developing new therapies for rare and ultra-rare diseases have unique challenges due to small patient populations, limited knowledge of disease biology and clinically relevant biomarkers. Batten disease, a fatal pediatric disease, is associated with the Cln gene family and Charles River is collaborating with Sanford Research to identify new clinically relevant endpoints for specific Batten disease variants.

In this podcast, Dr. Jill Weimer from Sanford Research and Dr. Antti Nurmi from Charles River discuss the importance of collaboration that combines scientific knowledge and cutting-edge technologies to accelerate the identification of novel translational biomarkers in Cln2 and Cln6 variants of Batten disease.


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