Research

While some may see Ellie’s future as bleak, her family and friends are full of hope. Currently, Ellie is given a mix of antioxidants and vitamins – but there are no definitive long-term therapies.

Dr. Ali Fatemi, Director of the Moser Center for Leukodystrophies at the Kennedy Krieger Institute in Baltimore, Maryland launched a research project in 2016 that will provide a road map toward long term therapies for LBSL. The outcomes of the research will become a very powerful tool for testing new, effective medications.

Their goal is to bring together highly complementary expertise from basic molecular methods, cell (and stem cell) culture, animal research, clinical outcome measure research, and machine learning methodologies through to clinical trial design.

The Kennedy Krieger team has received support from the LBSL community, the National Institutes of Health, and other internal sources to establish, to the best of our knowledge, the most comprehensive LBSL research effort in the world.

October 2018:
Moser Center for Leukodystrophies LBSL Research Update for Patients and Families

CLINICAL TRIALS

NEW CLINICAL TRIAL FOR LBSL