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Treatment with UX111 Reduced Cerebrospinal Fluid (CSF) Heparan Sulfate (HS) Exposure and Stabilized or Improved Functioning Across Dose, Age, and Stage of MPS IIIA

Author:Lau, Heather   Patra, Kaushik   Wolf, Melissa   Smith, Nicholas   Couce, María Luz   Rajan, Deepa   Truxal, Kristen   Lopez, María Jose   Fuller, Maria   Monteagudo, Eines   Dougherty, Lucy   del Toro, Mireia   Flanigan, Kevin   

Session Name:S19: Emerging Therapies in Child Neurology  

Topic:Child Neurology and Developmental Neurology  

Program Number:S19.009  

Author Institution:Ultragenyx Pharmaceutical Inc, CA  Women's and Children's Hospital and the University of Adelaide, Australia  Hospital Clínico Universitario de Santiago de Compostela, Santiago de Compostela, Spain  Childrens Hospital of Pittsburgh, Pittsburgh, PA  Nationwide Children'S Hospital, Columbus, OH  Hospital Clínico Universitario de Santiago de Compostela, Ramsbottom, Spain  Adelaide Medical School, North Adelaide, Australia  The Health Research Institute of Santiago de Compostela, Santiago de Compostela, Spain  Hospital Universitari Vall d’Hebron, Barcelona, Spain  Hospital Universitari Vall d’Hebron, Barcelona 08035, Spain  

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Real Life Effectiveness and Tolerability of Cladribine Tablets (CladT) after Five Years of Treatment

Author:Pato Pato, Antonio   Lorenzo González, José Ramón   García Estévez, Daniel Apolinar   López Real, Ana María   Rodriguez Regal, Ana   Costa Arpin, Eva   Rodriguez Rodriguez, María   Alvarez Rodriguez, Elena   Alberte Woodgard, Miguel   Aguado Valcarcel, Marta   González Suarez, Inés   Prieto Gonzalez, José María Oscar   

Session Name:P4: Multiple Sclerosis: Health Services/Outcomes Research and Real-world Evidence 4  

Topic:Multiple Sclerosis  

Program Number:P4.004  

Author Institution:Complexo Hospitalario de Vigo, Vigo, Spain  Hospital Povisa de Vigo, Vigo, Spain  Complexo Hospitalario de Ourense, Ourense, Spain  Complexo Hospitalario de A Coruña, A Coruña, Spain  Complexo Hospitalario de Pontevedra, Pontevedra, Spain  Complexo Hospitalario de Ferrol, Ferrol, Spain  Hospital Universitario Lucus Augusti, Lugo, Spain  Hospital Clínico Universitario de Santiago de Compostela, Santiago de Compostela, Spain  

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