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Real-world Outcomes of Nusinersen or Onasemnogene Abeparvovec (OA) Monotherapy, or Switching to OA from Nusinersen in SMA Patients Aged =6 Months

Author:Dabbous, Omar   Yang, Min   Georgieva, Mihaela   Toro, Walter   LaMarca, Nicole   Patel, Anish   Anderson, Annika   Akbarnejad, Hanane   Reyna, Sandra P.   

Session Name:P7: Child Neurology and Developmental Neurology: Spinomuscular Atrophy/Muscular Dystrophies  

Topic:Child Neurology and Developmental Neurology  

Program Number:P7.001  

Author Institution:Novartis Gene Therapies, Inc., Bannockburn, IL  Analysis Group, Inc., Boston, MA  Analysis Group, Inc., New York, NY  

Clinical Management of Rett Syndrome in the Real World: A Physician Survey

Author:Neul, Jeffrey   May, Damian   Satija, Ambika   Cheng, Wendy   Lema, Neema   Boca, Andra   Lefebvre, Patrick   Piña-Garza, Jesús Eric   

Session Name:P12: General Neurology: Neurogenetics 2  

Topic:General Neurology  

Program Number:P12.002  

Author Institution:Vanderbilt University Medical Center, Nashville, TN  Acadia Pharmaceuticals Inc., San Diego, CA  Analysis Group, Inc., Boston, MA  Analysis Group, Inc., Menlo Park, CA  Analysis Group, Inc., Montréal, QC, Canada  Centennial Children’s Hospital, Nashville, TN