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Topics

Learn about Neuronal Ceroid Lipofuscinosis type 2 (CLN2) topics

Safety and Efficacy Profile

Presentation

Topics

Safety and Efficacy Profile

Improved Developmental Outcomes with Early Initiation of Cerliponase Alfa Treatment in Children with CLN2 Disease

Seizures and Movement Disorders in Patients With CLN2 Disease Treated With Cerliponase Alfa in the Real-World Setting

Seizures and movement disorders in patients with CLN2 disease treated with cerliponase alfa in the real-world setting

Cerliponase alfa for the treatment of CLN2 disease in a patient cohort including children under 3 years of age

Seizures and movement disorders in patients with CLN2 disease treated with cerliponase alfa in the real-world setting

Cerliponase alfa for the treatment of CLN2 disease in a patient cohort including children under 3 years of age

Cerliponase alfa for the treatment of CLN2 disease in a patient cohort including children <3 years old

Real-world clinical outcomes of intraventricular cerliponase alfa in CLN2 disease: Comparison with a historical cohort

Seizures in Children with CLN2 Disease Receiving Cerliponase Alfa for >5 Years

Long-Term Treatment with Intracerebroventricular Cerliponase Alfa for Children with CLN2 Disease: Safety and Efficacy after >5 Years

Long-Term Treatment with Intracerebroventricular Cerliponase Alfa for Children with CLN2 Disease: Safety and Efficacy after >5 Years

Cerliponase alfa for the treatment of CLN2 disease in an expanded patient cohort including children younger than three years: Interim results from an ongoing clinical study

Cerliponase Alfa for the Treatment of CLN2 Disease in an Expanded Patient Cohort Including Children Younger than Three Years: Interim Results from an Ongoing Clinical Study

Genetic Testing and Diagnosis

Gene panels for skeletal dysplasia and epilepsy: Maximizing clinical utility through careful design, regular review, and clinician-laboratory collaboration

Clinical Utility of a Sponsored Gene Panel Testing Program for Pediatric Epilepsy and CLN2 Disease Diagnosis: Results from 14,589 Tests

Diagnostic yield and clinical utility of genetic testing in children with seizure onset after two years of age: Update over a 3-year program in Europe and the Middle East

Update on a Sponsored No-cost Epilepsy Gene Panel for Seizure Onset Between 2-4 Years of Age: Results from 682 Tests

Real World Evidence

Seizures and Movement Disorders in Patients With CLN2 Disease Treated With Cerliponase Alfa in the Real-World Setting

Seizures and movement disorders in patients with CLN2 disease treated with cerliponase alfa in the real-world setting

Seizures and movement disorders in patients with CLN2 disease treated with cerliponase alfa in the real-world setting

Real-world clinical outcomes of intraventricular cerliponase alfa in CLN2 disease: Comparison with a historical cohort

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