NCL2018, the 16th International Congress on Neuronal Ceroid Lipofuscinosis (Batten Disease)

Massachusetts General Hospital, Boston MA

Investigators

Abstract

PROJECT SUMMARY The group of disorders known as the neuronal ceroid lipofuscinoses (NCLs), also known as Batten disease, are collectively the most common cause of childhood onset neurodegeneration in the U.S. and worldwide. Major recent advances in disease pathogenesis and in therapy development have driven the field towards an exciting translational stage. The increasing application of genomics to diagnosis of rare disease has further highlighted overlap with common disease, such as the overlap observed between NCL and non-syndromic retinal disease, Parkinsonism, frontotemporal dementia, and progressive myoclonic epilepsy without lysosomal storage, which has broadened the field of researchers and clinicians with an interest in the NCLs. This past year, the NCL community has seen high impact success with the first ever FDA-approved therapy for any form of NCL. An ?International Congress on Neuronal Ceroid Lipofuscinosis (Batten disease)? is held approximately every two years, and it is the only scientific meeting that convenes basic researchers, clinicians, families and patient advocacy representatives focused on NCL. There is a critical need to continue this highly successful meeting with the aim of further advancing research and translation towards therapies for all forms of NCL. The next meeting, NCL2018, will be held at the Royal Holloway College, University of London, September 12-16, 2018. At this year's meeting, we will organize a scientific agenda with the highest standard of scientific and clinical presentations, and we will present the same information in a format that is accessible to NCL families. This application is intended to support the conference participation of trainees and junior level investigators from US-based institutions.