Factual Friday

Did you know ALS and Batten disease are both neurodegenerative diseases? In fact, Batten is the most common pediatric neurodegenerative disorder. 

Chris Martin of CAVU Media Works took on the #ALSicebucketchallenge with a twist. Thank you for helping raise awareness!

BBDF’s Principal Scientist Speaks at the Rare Disease Collaboration Summit

PhiladelphiaDanielle Kerkovich, PhD, traveled to Philadelphia, PA, to speak at the Rare Disease Collaboration Summit hosted by ExL Pharma on August 12-13, 2014. Dr. Kerkovich’s talk was entitled “Extra Levels of Care:  Working with Patients, Parents, Caregivers, and Review Boards on the Development of Orphan Drugs for Pediatric Indications.” The invitation was sparked by recognition of BBDF’s close connection to other Batten and brain disorders organizations. The Rare Disease Collaboration Summit harnesses the collective resources of industry and patient groups into creating working-group relationships that shape legislation and accelerate regulatory approval. It builds lasting partnerships with patient advocacy groups in order to construct more flexible and patient-centric clinical trial protocols and accelerate the regulatory approval of orphan drugs. The summit was incredibly informative and Danielle brings that knowledge back with her to the Foundation.