The group of fatal inherited 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. Despite the identification of many of the disease-causing gene mutations, progress towards developing effective therapies has been hampered by a relatively poor understanding of the pathogenesis of these disorders. The scientific and translational landscape of these disorders has changed dramatically in recent years with the first ever FDA-approved therapy (enzyme replacement for CLN2 disease), and clinical trials of gene therapy in progress for several other forms of NCL after very promising pre-clinical studies. These advances are underpinned by new disease models, a much better understanding of disease mechanisms, and the cell biology of the proteins that are deficient in these disorders. An ?International Congress on Neuronal Ceroid Lipofuscinosis (Batten disease)? is held approximately every two years. With recent significant basic science and translational developments within this field there is a critical need to continue this highly successful series of conferences, with the aim of further advancing research and translation towards effective therapies for all forms of NCL. Indeed, this is only scientific meeting that convenes basic researchers, clinicians, families and patient advocacy representatives focused on the NCLs. The next meeting, NCL2020, will be held at Washington University in St Louis, October 7-11, 2020. At this meeting, we will organize a scientific agenda with the highest standard of scientific and clinical presentations, and we will also present the same information in a format that is accessible to NCL families. In addition, we are fully committed to support and ensure the appropriate representation of women, minority scientists, persons with disabilities and to facilitate more junior investigators (including students) to attend and participate in this meeting. This application is intended to support the conference participation of trainees and junior level investigators from US-based institutions.
The 17th International Congress on NCL is focused on the group of fatal childhood neurodegenerative disorders known as neuronal ceroid lipofuscinosis (NCL) or Batten disease. The scientific and translational landscape of these disorders has changed dramatically in recent years, with better understand of disease mechanisms, the first ever FDA-approved therapy and several clinical trials in progress. This is the only scientific conference that is dedicated to the NCLs and serves a critical need in bringing together researchers, clinicians, patient support groups and affected families.
