The 59th Annual Meeting of the Japanese Society of Child Neurology

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metabolic diseases

[S15] Symposium 15
Recent Advancement of Inherited Metabolic Diseases: Treatment of Neuronal Ceroid Lipofuscinosis(NCL)

Fri. Jun 16, 2017 2:10 PM - 4:10 PM Room No.7 (10F Conference Room 1004-1005)

Chair:Akira Oka(The University of Tokyo Review Committee of The Japan Obstetric Compensation System for Cerebral Palsy), Norio Sakai(Division of Health Science, Osaka University Graduate School of Medicine)

[S15-2] 2）Intracerebroventricular cerliponase alfa（BMN 190）in children with CLN2 disease：Results from a phase 1／2, open—label, dose—escalation study

○Nicola Specchio1,Angela Schulz2, Paul Gissen3, De los Emily Reyes4, Ruth Williams5, Heather Cahan6, Peter Slasor6, David Jacoby6 (1.Bambino Gesu Childrens Hospital, IRCCS, Rome, Italy, 2.University Medical Center Hamburg-Eppendorf, Hamburg, Germany, 3.Great Ormond Street Hospital for Children, London, United Kingdom, 4.Nationwide Childrens Hospital, The Ohio State University, Columbus, OH, United States, 5.Guys and St Thomas NHS Foundation Trust, London, United Kingdom, 6.BioMarin Pharmaceutical Inc., Novato, CA, United States)

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