Oral Presentation International Congress on Neuronal Ceroid Lipofuscinoses 2025

Improved developmental outcomes with early initiation of cerliponase alfa treatment in children with CLN2 disease (126420)

Angela Schulz 1 2 , Emily de Los Reyes 3 , Paul Gissen 4 , Nicola Specchio 5 , Kathleen Delaney 6 , Jessica Cohen Pfeffer 6 , Pascal Reisewitz 6
  1. University Medical Center Hamburg-Eppendorf, Hamburg, Germany
  2. German Center for Child and Adolescent Health, partner site Hamburg, Hamburg, Germany
  3. Nationwide Children's Hospital, Columbus, Ohio, USA
  4. NIHR Great Ormond Street Hospital Biomedical Research Centre, University College, London, UK
  5. Bambino Gesu Children's Hospital, IRCCS, Rome, Italy
  6. BioMarin Pharmaceutical Inc, Novato, California, USA
Publish consent withheld