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Correction of Duchenne Muscular Dystrophy Mutations by CRISPR/Cas9 Gene Editing Using Homology-Independent Targeted Integration

Author:Nicolau, Stefan   Vetter, Tatyana   Stephenson, Anthony   Dufresne, Gabrielle   Frair, Emma   Trecha, Breanna   Sarff, Jessica   Flanigan, Kevin   

Session Name:P7: Child Neurology and Developmental Neurology: Neuromuscular 2  

Topic:Child Neurology and Developmental Neurology  

Program Number:P7.006  

Author Institution:Nationwide Children's Hospital, Columbus, OH  

Study Design of Subcutaneous Efgartigimod PH20 in Juvenile Generalized Myasthenia Gravis

Author:Schwaede, Abigail   Kuntz, Nancy   Bogatyreva, Anna   Menezes, Flavia   Giacobbe, Juliette   Van Bragt, Tonke   Kostera-Pruszczyk, Anna   Ramdas, Sithara   

Session Name:P7: Child Neurology and Developmental Neurology: Neuromuscular 2  

Topic:Child Neurology and Developmental Neurology  

Program Number:P7.009  

Author Institution:Ann & Robert H Lurie Children's Hospital of Chicago, Chicago, IL  Medical University of Warsaw, Warsaw, Poland  

The Urgent Need for Evidence Over Anecdote: Equipoise in the ExTINGUISH Clinical Trial in Anti-NMDAR Encephalitis

Author:Hoshina, Yoji   Smith, Tammy   Wong, Ka-Ho   Peterson, Lisa   Zekeridou, Anastasia   Aboseif, Albert   Coffey, Christopher   Wright, Melissa   Banwell, Brenda   Dialino-Felix, Annalisa   FLAVIN, SUSAN   Dill, Lisa   Cho, Hyun   Dalmau, Josep   Titulaer, Maarten   Day, Gregory   Clardy,   

Session Name:P7: Autoimmune Neurology: NMOSD and MOGAD 1  

Topic:Autoimmune Neurology  

Program Number:P7.016  

Author Institution:University of Utah, Salt Lake City, UT  ARUP Laboratories, Salt Lake City, UT  Neuroimmunology Laboratory, Mayo Clinic, Rochester, MN  Mayo Clinic Rochester, Rochester, MN  University of Iowa, Iowa City, IA  Childrens Hospital of Philadelphia, Philadelphia, PA  Amgen Inc., Spring City, CA  NIH, Bethesda, MD  University of Barcelona, Barcelona, Spain  Erasmus Medical Center, Rotterdam, Netherlands  Mayo Clinic, Jacksonville, FL  

Febrile Status Epilepticus developing in refractory Kawasaki disease without meningoencephalitis triggered by Parvovirus B19 infection

Author:Khan, Zain   Kakawand, Soroush   Thomas, Laura   

Session Name:P7: Child Neurology and Developmental Neurology: Neuro-immunology and Neuro-infectious 1  

Topic:Child Neurology and Developmental Neurology  

Program Number:P7.019  

Author Institution:Oklahoma University Health Sciences Center, Oklahoma City, OK  University of Oklahoma Health Sciences Center, Oklahoma City, OK  Oklahoma Children's Hospital, Oklahoma City, OK  

Real-World Experiences with VMAT2 Inhibitors in Pediatric Hyperkinetic Movement Disorders

Author:Manohar, Sujal   Berger, Rebecca   Jacobe, Jennifer   Hull, Mariam   

Session Name:P7: Child Neurology and Developmental Neurology: Neuro-immunology and Neuro-infectious 1  

Topic:Child Neurology and Developmental Neurology  

Program Number:P7.020  

Author Institution:Baylor College of Medicine, Houston, TX  Texas Children's Hospital, Pediatric Neurology, Houston, TX