Thursday, 30th October International Congress on Neuronal Ceroid Lipofuscinoses 2025

All Speakers from the session will take part in the Q&A panel

• ESRRA: a new druggable target to tackle Batten disease - Miriam MA Aurilia
• Adenine Base Editors: A Novel Approach to Treat CLN2 Disease - Jill M Weimer
• Investigating the pathophysiological basis of dysphagia in CLN1, CLN2, and CLN3 mouse models and the therapeutic effects of gene therapy - Ewa A Ziółkowska
• Treatment of CLN1 with TSHA-118 for AAV-9-Mediated PPT1 Gene Transfer Through an Expanded Access Program - Elizabeth Berry-Kravis

All Speakers from the session will take part in the Q&A panel

• Adaptive Behavior in CLN3 disease and relationship to disease stage - Heather R Adams
• A Flupirtine Benzyl Carbamate Derivative Mitigates Motor/Cognitive Deficits and Improves Molecular Pathology in the Cln6^nclf Mouse - Rose-Mary Boustany
• Therapeutic Evaluation of Trehalose Infusions in CLN5 and CLN6 Affected Sheep Models - Jazmine Brash
• Cardiolipin and NCL: Investigating phospholipid alterations in ovine models. - Ella M J Harris
• International Models of Care for Childhood Dementia: An Investigation to Inform the Australian Context - Gail Hilton
• Peripheral pathology in sheep with CLN5 and CLN6 Batten disease - Samantha Murray
• Zebrafish model of CLN2 disease provides a platform for high-throughput drug screening and identifies pregnenolone as a hit compound with therapeutic potential - Claire Russell
• Behavioral Phenotype and Symptoms of Autism Spectrum Disorder in Young Children with CLN2 Batten Disease - Jessica F Scherr
• Longitudinal analysis of anti-drug antibody response against Cerliponase alfa in CLN2 patients - Lena Marie Westermann
• Analysis of brain histopathology following pregnenolone treatment in CLN2 disease mice - Ewa A Ziółkowska
• Advancing Therapeutic Strategies for CLN3 Batten Disease: iPSC-Based Models and Focused Ultrasound for Drug Delivery Across the Blood-Brain Barrier - Juliana CS Chaves

• From cell phenotypes to candidate therapies: high-content screening in CLN3 disease - Llinos Honeybun
• CLN3 patient-derived organoids as preclinical platforms for drug screening and gene therapy testing - Magnar Bjørås
• Deep, Untargeted Multi-omics Biomarker Landscape in Large Animal Models of CLN1, -2, -3, -5,and -6 Batten Disease - Clarissa D Booth
• CLN2 Batten Disease biomarker profiling reveals cardiovascular-associated metabolic dysfunction - Joelle T Anderson

All Speakers from the session will take part in the Q&A panel

• Speech, Language and Non-verbal Communication in CLN2 and CLN3 Batten Disease - Lottie D Morison
• Urinary Glycerophosphoinositol is a potentially non-invasive clinically applicable biomarker for CLN3 disease - Wendy E Heywood
• Assessment of cognitive decline in CLN3 using the Vineland Adaptive Behavior Scale and the CLN3 Disease Staging System - Miriam Nickel

All Speakers from the session will take part in the Q&A panel

Be ready for bus departure at 17:40
Pre-registration for the dinner is essential