Neuronal ceroid lipofuscinosis 3
- Juvenile neuronal ceroid lipofuscinosis
- Vogt Spielmeyer disease
- Spielmeyer Sjogren disease
- CLN3 disease, juvenile
News & Events
Batten Disease May Benefit From Gene Therapy
November 13, 2015
NCATS Co-Sponsored Conferences
Gordon Research Conference and Gordon Research Seminar on Lysosomes and Endocytosis, Sunday, June 15, 2014 - Friday, June 20, 2014
Location: Proctor Academy, Andover, NH
Description: The main goal of the Lysosomes and Endocytosis GRC is to foster the dissemination of current research results and the establishment of new research areas and new collaborations in the area of the cell biology of endocytosis, lysosomes, endosomes and related organelles. We hope that many of these new directions and collaborations will be directed toward the etiology, diagnosis and treatment of rare genetic diseases such as lysosomal storage disorders, Hermansky-Pudlak syndrome, Chediak-Higashi syndrome, Niemann Pick disease and tuberous sclerosis, among others.
Outcome Measures and Infrastructure for Phase III Studies in Batten Disease, JNCL, Saturday, September 22, 2012 - Monday, September 24, 2012
Location: Hyatt Regency, Atlanta, GA
Description: The main objective of the meeting is to bring together clinical research experts in JNCL to focus, for the first time, on establishing common ground for outcomes and infrastructure in support of Phase III Clinical Trials. Towards this end, it is anticipated that at the end of the meeting the participants will be able to identify potential clinical trial endpoints for Juvenile Neuronal Ceroid Lipofuscinosis (JNCL, CLN3 disease, Batten Disease); build upon the infrastructure for an International Patient Registry for JNCL and other NCL diseases, and provide opportunities for junior investigators pursuing research in JNCL to learn about and participate in the clinical research development process.
Third Genome Dynamics in the Neurosciences Conference, Sunday, July 18, 2010 - Thursday, July 22, 2010
Location: The Hilton Metropole, Brighton, England
Description: The goal of this meeting was to integrate basic processes of DNA damage signaling and repair and clinical aspects of neurological and neurodegenerative disease. The program was designed to bring together leading scientists with primary interests in DNA damage signaling with those working in specific related neurodegenerative disease areas as a means for integrating these fields. It was anticipated that this would generate insights into how normal processes of genome maintenance in the brain contribute to the prevention of a wide range of diseases.