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Long-term Follow-up of Cipaglucosidase Alfa/Miglustat in Ambulatory Patients with Pompe Disease: An Open-label Phase I/II Study (ATB200-02)

Author:Mozaffar, Tahseen   Schoser, Benedikt   Kishnani, Priya   Bratkovic, Drago   Clemens, Paula R.   Goker-Alpan, Ozlem   Ming, Xue   Roberts, Mark   Sivakumar, Kumaraswamy   Van Der Ploeg, Ans   Goldman, Mitchell   Wright, Jacquelyn   Holdbrook, Fred   Jain, Vipul   Sitaraman, Sheela   Wasfi, Yasmine   Byrne, Barry   

Session Name:S48: Therapeutics for Muscle Disease  

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:S48.007  

Author Institution:University of California, Irvine, Irvine, CA  Friedrich-Baur-Institut, Neurologische Klinik, Ludwig-Maximilians-Universität München, Munich, Germany  Duke University Medical Center, Durham, NC  PARC Research Clinic, Royal Adelaide Hospital, Adelaide, SA, Australia  Department of Neurology, University of Pittsburgh School of Medicine, Division Chief, Neurology, Medical Service Line, VA Pittsburgh Healthcare System, Pittsburgh, PA  Lysosomal and Rare Disorders Research and Treatment Center, Fairfax, VA  Department of Neurology, Rutgers New Jersey Medical School, Newark, NJ  Salford Royal NHS Foundation Trust, Salford, United Kingdom  Neuromuscular Clinic and Research Center, Phoenix, AZ  Erasmus MC University Medical Center, Rotterdam, Netherlands  Amicus Therapeutics, Inc., Philadelphia, PA  University of Florida, Gainesville, FL  

Ketogenic Diet Intervention Improves Sleep in Patients with Relapsing Multiple Sclerosis

Author:Perlman, Jacob   Lehner-Gulotta, Diana   Wetmore, Emma   Coleman, Rachael   Banwell, Brenda   Bergqvist, A. G.   Chen, Shanshan   Conaway, Mark   Goldman, Myla   Morse, Anne Marie   Brenton, James   

Session Name:P8: MS Therapeutics 2  

Topic:MS and Inflammatory Disease  

Program Number:P8.009  

Author Institution:University of Virginia Health System, Charlottesville, VA  Medical University of South Carolina, Charleston, SC  Children's Hospital of Philadelphia, Philadelphia, PA  Virginia Commonwealth University , Richmond, VA  University of Virginia School of Medicine, Charlottesville, VA  Virginia Commonwealth University, Richmond, VA  Geisinger Commonwealth School of Medicine, Scranton, PA  

Multiple Sclerosis & Family Planning: Patient Experience

Author:Kelly, Erin   Engel, Casey   Pearsall, Rylan   Goldman, Myla   

Session Name:P9: MS Special Populations  

Topic:MS and Inflammatory Disease  

Program Number:P9.006  

Author Institution:Virginia Commonwealth University School of Medicine, Richmond, VA  University of Virginia, Charlottesville, VA  VCU Health Department of Neurology, Richmond, VA  

Environmental Exposure Speeds Symptom Progression in PD

Author:Brown, Ethan   Goldman, Samuel   San Luciano, Marta   Korell, Monica   Tanner, Caroline   

Session Name:P6: Movement Disorders: Environmental Factors and Phenotyping Parkinsonisms 2  

Topic:Movement Disorders  

Program Number:P6.005  

Author Institution:University of California San Francisco, Weill Institute for Neurosciences, San Francisco, CA  University of California San Francisco, Occupational & Environmental Medicine, San Francisco, CA  

Efficacy of Omaveloxolone in Friedreich’s Ataxia: Post-Hoc Analysis Using Global Statistics Test to Strengthen Secondary Endpoint Analyses

Author:Hendrix, Suzanne   Goldsberry, Angie   Meyer, Colin   Lynch, David   

Session Name:P8: Movement Disorders: Ataxias 1  

Topic:Movement Disorders  

Program Number:P8.011  

Author Institution:Pentara Corporation, Salt Lake City, UT  Reata Pharmaceuticals Inc., Plano, TX  Children's Hospital of Philadelphia, Philadelphia, PA