Each year, BDSRA invites proposals from over 200 scientists working in the field to fund the most promising basic and translational (moving to clinical trial) research. Each proposal is peer-reviewed by experts in lysosomal diseases at universities, children’s hospitals and in the pharmaceutical industry to provide well-rounded reviews.
We are honored to partner with the following organizations who funded this year’s awardees: Drew’s Hope, ForeBatten, Haley’s Heroes Foundation, and Noah’s Hope/Hope 4 Bridget.
The 2019 BDSRA Research Award Winners
Dr. Heather Adams
From the University of Rochester Medical Center is working in CLN2 and CLN3 of Batten on a project entitled Characterizing sleep dysfunction in children with NCL disorders.
Dr. Elena Batrakova
From the University of North Carolina at Chapel Hill is working in CLN2 on a project entitled Novel Nanoformulation of TPP1 for Treatment of LINCL Batten Disease CLN2.
Dr. Thomas M Wishart
From the Roslin Institute, University of Edinburgh is working in CLN1 and CLN5 on a project entitled Using ovine models of CLN1 and CLN5 disease to identify molecular biomarkers of disease and therapeutic efficacy.
Dr. Milen Velinov
From the New York State Institute for Basic Research in Developmental Disabilities, is working in CLN4 of a project entitled Deferiprone iron chelation therapy to alleviate CLN4 pathology in patients with Autosomal Dominant Neuronal Ceroid Lipofuscinosis-Kufs Disease.