| Andrea Miele | Initiation of cerliponase alpha at 4-months as pre-symptomatic treatment of CLN2 Batten Disease |
| Jennifer Vermilion | Genotype-Phenotype Relationships in CLN3 Disease: Insights from a Natural History Study |
| Jennifer Vermilion | Clinical Symptoms in Late Infantile and Juvenile Onset CLN7 Disease |
| Miriam Nickel | Analysis of longitudinal natural history data in infantile CLN1 patients from two independent international cohorts using the Hamburg iCRS scale |
| Jennifer Vermilion | Impact of CLN3 Disease on Child Quality of Life and Family Function |
| Ineka T Whiteman | A Timeline of Symptom Onset and Disease Progression in CLN3 Disease |
| Joanna Nightingale | Impact of Treatment with Cerliponase Alfa on CLN2 Associated Symptoms - Evidence from the Families and Educational Professionals in the United Kingdom |
| Stefania Della Vecchia | Gastrointestinal Involvement in Neuronal Ceroid Lipofuscinoses: A Pilot Study on Symptom Burden and Quality of Life in a Pediatric Cohort |
PANEL MEMBERS
Sara Mole
Ineka Whiteman
Karen Kahn
Kasparas Petkevicius
Jon Cooper
| Ineka T Whiteman | The Lynchpin of Progress: The Role of Patient Advocates in Driving Rare Disease Research, Development and Access |
| Angela Schulz | Improved developmental outcomes with early initiation of cerliponase alfa treatment in children with CLN2 disease |
| Lena Marie Westermann | Real-world pre-symptomatic treatment in CLN2 disease: Learnings from families with multiple affected children |
| Catherine O. Jordan | Intravitreal Enzyme Replacement Therapy to Prevent Retinal Disease Progression in Children with Neuronal Ceroid Lipofuscinosis Type 2 (CLN2):Â Interim 3-Year Safety and Efficacy Outcomes |
| Christina Ohnsman | Two year results of first-in-human intracisternal TTX-181 investigational AAV9 gene therapy in a child with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2) |
| Christina Ohnsman | One-year results from the first-in-human clinical trial of TTX-381, an investigational gene therapy for the treatment of ocular manifestations of CLN2 Batten disease |
| Alexandra M Johnson | Outcomes of miglustat use in five children with CLN3 disease |
| Emily de los Reyes | AAV9 Gene Replacement Therapy for CLN3 Batten Disease Suggests Significant Slowing of Disease Progression and Maintenance of Speech, Cognitive and Motor Function at up to 5 Years Post Treatment |
Please join us for a group photo for NCL2025
Dinner will be at the Novotel Lagoon Beach
Pre-registration for the dinner is essential