PARALLEL SESSION 2D: Neurometabolic Disorders I
Tracks
MC 3 HALL
| Wednesday, September 18, 2019 |
| 4:30 PM - 6:15 PM |
| MC 3 HALL |
Speaker
Prof. Barbara Plecko
Department of General Paediatrics, University Clinic for Pediatrics and Adolescent Medicine, Graz Medical University
Biomarkers for a quick diagnosis
4:30 PM - 4:55 PM
Dr. Caroline Sevin
Hôpital Universitaire Bicêtre-Paris Sud
OC036 | Phase 2/3 trial to assess the safety and efficacy of Lenti-D Hematopoietic Stem Cell Gene Therapy for Cerebral Adrenoleukodystrophy
Dr. Samuel Groeschel
University Children's Hospital Tübingen
OC037 | Effect of intrathecal recombinant human Arylsulfatase A Enzyme Replacement Therapy on structural brain MRI in children with Metachromatic Leukodystrophy
Dr. Francesca Fumagalli
San Raffaele Scientific Institute
OC038 | Long term effect of Lentiviral (LV) Hematopoietic Stem Cell Gene Therapy (HSC-GT) on the nervous system in early-onset Metachromatic Leukodystrophy (MLD)
Dr. Florian Eichler
Leukodystrophy Service, Department of Neurology, MassGeneral Hospital for Children
OC039 | An observational study of outcomes of Allogeneic Hematopoietic Stem Cell Transplant in patients with Cerebral Adrenoleukodystrophy (CALD)
Dr. Angela Schulz
University Medical Center Hamburg-Eppendorf
OC040 | 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
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Discussion
6:10 PM - 6:15 PMChair
Jaume Campistol Plana
Barcelona University, Neurology Department, Hospital Sant Joan de Déu
Helen Michelakakis
Department of Enzymology and Cellular Function, Institute of Child Health
