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

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English Session

metabolic diseases

[E1] English Session 1
neurodegenerative disease / metabolic disease

Thu. Jun 15, 2017 11:20 AM - 12:20 PM Room No.2 (6F Sub Hall)

Chair:Akira Oka （Department of Pediatrics, The University of Tokyo, Tokyo, Japan） Norio Sakai（Division of Health Science, Osaka University Graduate School of Medicine, Suita, Japan）

[E-001] Intracerebroventricular cerliponase alfa in children with CLN2 disease：a Phase 1／2 study

○Specchio Nicola1, 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 Children s 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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