# NCL2020, The 17th International Congress on the Neuronal Ceroid Lipofuscinoses (NCLs) or Batten Disease

> **NIH NIH R13** · WASHINGTON UNIVERSITY · 2020 · $15,000

## Abstract

PROJECT SUMMARY
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.

## Key facts

- **NIH application ID:** 9993731
- **Project number:** 1R13NS117015-01
- **Recipient organization:** WASHINGTON UNIVERSITY
- **Principal Investigator:** JONATHAN D COOPER
- **Activity code:** R13 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $15,000
- **Award type:** 1
- **Project period:** 2020-04-01 → 2022-03-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9993731

## Citation

> US National Institutes of Health, RePORTER application 9993731, NCL2020, The 17th International Congress on the Neuronal Ceroid Lipofuscinoses (NCLs) or Batten Disease (1R13NS117015-01). Retrieved via AI Analytics 2026-06-12 from https://api.ai-analytics.org/grant/nih/9993731. Licensed CC0.

---

*[NIH grants dataset](/datasets/nih-grants) · CC0 1.0*