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Motor Outcomes to Validate Evaluations in Facioscapulohumeral muscular dystrophy (MOVE FSHD): Preliminary Baseline Characteristics

Author:Walker, Michaela   Butterfield, Russell   Day, John   Eichinger, Katy   Elsheikh, Bakri   Friedman, Seth   Genge, Angela   Higgs, Kiley   Johnson, Nicholas   Jones, Peter   Leung, Doris   Lewis, Leann   Lochmuller, Hanns   O'Ferrall, Erin   Martens, William   Shaw, Dennis   Shieh, Perry   Subramony, S   Trivedi, Jaya   Wang, Leo   Wicklund, Matthew   Tawil, Alrabi   Statland, Jeffrey   

Session Name:S7: Outcome Measures and Observational Studies of Neuromuscular Diseases  

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:S7.004  

Author Institution:University of Kansas Medical Center, Fairway, KS  University of Utah, Salt Lake City, UT  Stanford University School of Medicine, Palo Alto, CA  University of Rochester Medical Center, Rochester, NY  The Ohio State University Wexner Medical Center, Columbus, OH  Seattle Children's Hospital , Seattle, WA  Mcgill University, Montreal, QC, Canada  University of Kansas Medical Center, Kansas City, KS  Virginia Commonwealth University, Richmond, VA  University of Nevada Reno, Reno, NV  Kennedy Krieger Institute, Baltimore, MD  Children's Hospital of Eastern Ontario & Ottawa Hospital Research Institute , Ottawa, ON, Canada  McGill University & Montreal Neurological Institute, Montreal, QC, Canada  Seattle Children's Hospital, Seattle, WA  University of California Los Angeles, Los Angeles, CA  University of Florida, Gainesville, FL  University of Texas Southwestern Medical Center, Dallas, TX  University of Washington, Seattle, WA  University of Colorado, Aurora, CO  

The FSHD Composite Outcome Measure (FSHD-COM) is Reliable, Valid, and Measures Disease Progression

Author:Eichinger, Katy   McDermott, Michael   Higgs, Kiley   Walker, Michaela   Lewis, Leann   Martens, William   Leung, Doris   Stinson, Nikia   Holzer, Megan M   Sacconi, Sabrina   Garcia, Jeremy   De Paz Benito, Victor   Mul, Karlien   Sansone, Valeria   Carraro, Elena   Becchiati, Stefano   Frisoni, Maria Chiara   Wang, Leo   Kieu, Catherine   Shieh, Perry   Skura, Christy   Elsheikh, Bakri   Kelly, Kristina   Jaworek, Andrea   LoRusso, Samantha   Butterfield, Russell   Wilson, Amelia   McIntyre, Melissa   Johnson, Nicholas   Butler, Amanda   Jones, Aileen   Hayes, Melissa   Baker, Lindsay   Sasidharan, Sandhya   Tawil, Alrabi   Statland, Jeffrey   

Session Name:S7: Outcome Measures and Observational Studies of Neuromuscular Diseases  

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:S7.005  

Author Institution:University of Rochester Medical Center, Rochester, NY  University of Kansas Medical Center, Kansas City, KS  University of Kansas Medical Center, Fairway, KS  Kennedy Krieger Institute, Baltimore, MD  Nice University, Nice, France  Radboud University, Nijmegen, Netherlands  The NEMO Clinical Center - Neurorehabilitation Unit, University of Milan, Milan, Italy  University of Washington, Seattle, WA  UCLA, Los Angeles, CA  The Ohio State University Wexner Medical Center, Columbus, OH  Kaiser, Lone Tree, CO  University of Utah, Salt Lake City, UT  Virginia Commonwealth University, Richmond, VA  

Annualized Rates of Change from A Phase 2, Randomized, Double-Blind, Placebo-Controlled, 48-Week Study of Losmapimod in Subjects with FSHD: ReDUX4

Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamic Effects of AOC 1020 Administered Intravenously to Adult Patients with Facioscapulohumeral Muscular Dystrophy (FORTITUDE) Trial Design