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Inverting the Drug Discovery Funnel: In Vivo First Screening for mAb Blood-brain Barrier Penetration Yields Molecules with Novel Uptake Properties

Defining the Neuron-specific Mechanisms of WIPI4 and Autophagy Dysfunction in ß-propeller Protein-associated Neurodegeneration

NEO1/NEO-EXT Studies: Long-term Muscle Quantitative Magnetic Resonance Imaging and Functional Efficacy in Adults With Late-onset Pompe Disease (LOPD) on Avalglucosidase Alfa Treatment

Author:Ladha, Shafeeq   Carlier, Pierre   Vissing, John   Dimachkie, Mazen   Byrne, Barry   Barohn, Richard   Kishnani, Priya   Mengel, Eugen   Sacconi, Sabrina   Trivedi, Jaya   Young, Peter   an Haack, Kristina   Armstrong, Nicole   Miossec, Patrick   Thibault, Nathan   Sparks, Susan   Schoser, Benedikt   Diaz-Manera, Jordi   

Session Name:P8: General Neurology: Assorted Neurologic Basic Science  

Topic:General Neurology  

Program Number:P8.010  

Author Institution:Gregory W. Fulton ALS and Neuromuscular Center, Barrow Neurological Institute, Phoenix, AZ  Université Paris-Saclay, CEA, DRF, Service Hospitalier Frédéric Joliot, Orsay, France  Copenhagen Neuromuscular Center, Rigshospitalet, University of Copenhagen, Copenhagen, Denmark  University of Kansas Medical Center, Kansas City, KS  University of Florida, Gainesville, FL  University of Missouri, Columbia, MO  Duke University Medical Center, Durham, NC  SphinCS GmbH, Institute of Clinical Science for LSD, Hochheim, Germany  Neuromuscular Diseases Centre, University Hospital of Nice (CHU), Nice, France  University of Texas Southwestern Medical Center, Dallas, TX  Medical Park Bad Feilnbach, Bad Feilnbach, Germany  Sanofi, Chilly-Mazarin, France  Sanofi, Cambridge, MA  Friedrich-Baur-Institute, Ludwig-Maximilians-University Klinikum München, Munich, Germany  John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle, United Kingdom