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Beyond Motor Function in Adults With Spinal Muscular Atrophy (SMA): A Systematic Literature Review of Real-world Outcomes With Risdiplam

Author:Guittari, Carol Jean   Gorni, Ksenija   Sideris, Eleftherios   Davies, Evan   Mahajan, Anadi   Kumari, Sandhya   Simpson, Alex   Sutherland, C. Simone   

Session Name:P5: Neuromuscular and Clinical Neurophysiology (EMG): Motor Neuron Disorders  

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:P5.001  

Author Institution:PDMA Neuroscience and Rare Disease, Genentech, Inc., South San Francisco, CA, USA, South San Francisco, CA  PDMA Neuroscience and Rare Disease, F. Hoffmann-La Roche Ltd, Basel, Switzerland, Switzerland  Global Access, F. Hoffmann-La Roche Ltd, Basel, Switzerland, Switzerland  Bridge Medical Consulting Ltd., London, UK, United Kingdom  

Real-world Motor Function Outcomes with Risdiplam in Types 2 and 3 Spinal Muscular Atrophy (SMA): A Systematic Literature Review and Meta-analysis

Author:Guittari, Carol Jean   Davies, Evan   Sutherland, C. Simone   Gorni, Ksenija   Edwards, Susan   Kjærgaard, Maya   Mahajan, Anadi   Gupta, Digant   Khan, Mudasir   Kumari, Sandyha   Sideris, Eleftherios   

Session Name:P5: Neuromuscular and Clinical Neurophysiology (EMG): Motor Neuron Disorders  

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:P5.002  

Author Institution:Genentech Inc, CA, USA, South San Francisco, CA  Global Access, F. Hoffmann-La Roche Ltd, Basel, Switzerland, Switzerland  PDMA Neuroscience and Rare Disease, F. Hoffmann-La Roche Ltd, Basel, Switzerland, Switzerland  Pharma Development, Data Sciences, F. Hoffmann-La Roche Ltd, Basel, Switzerland, Switzerland  Roche Pharmaceuticals AS, Copenhagen, Denmark, Denmark  Bridge Medical Consulting Ltd, London, UK, United Kingdom  

Exploration of the Diagnostic and Treatment Journey of Adult-onset (Type Four) SMA

Author:Eisenman, Lauren   Curry, Mary   Glascock, Jacqueline   

Session Name:P5: Neuromuscular and Clinical Neurophysiology (EMG): Motor Neuron Disorders  

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:P5.003  

Author Institution:Cure SMA, Schaumburg, IL  

Exploration of Patient and Caregiver Burden Due to Insurance Denials and Appeals for Spinal Muscular Atrophy (SMA) Treatment and Healthcare Access

Author:Eisenman, Lauren   Curry, Mary   Belter, Lisa   Friesz, Maynard   Schroth, Mary   

Session Name:P5: Neuromuscular and Clinical Neurophysiology (EMG): Motor Neuron Disorders  

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:P5.004  

Author Institution:Cure SMA, Schaumburg, IL  

Long-term Efficacy and Safety of Risdiplam in Adults With 5q Spinal Muscular Atrophy (SMA): A Large Prospective Multi-centre Observational Study

Author:Hewamadduma, Channa   Yeo, Jing Ming   Street, Jon   Nevin, Katie   Accad, Grace   Russell, Jean   

Session Name:P5: Neuromuscular and Clinical Neurophysiology (EMG): Motor Neuron Disorders  

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:P5.005  

Author Institution:Sheffield Institute for Translational Neurosciences (SITRAN), University of Sheffield, Sheffield, United Kingdom  Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, United Kingdom  Sheffield Teaching Hospitals NHS Foundation Trust, United Kingdom  University of Sheffield, United Kingdom