Deramiocel Significantly Slows Upper Limb Functional Decline in Duchenne Muscular Dystrophy: Skeletal Muscle Outcomes from the Phase 3 HOPE-3 Trial

Author:Iannaccone, Susan   McDonald, Craig   Veerapandiyan, Aravindhan   Varadhachary, Arun   Ramos-Platt, Leigh   Gambetta, Kathryn   Butterfield, Russell   Leffler, Mindy   maharry, kati   Hogan, Nathaniel J.   apkon, susan   Tian, Cuixia   Mathews, Katherine   Bass, Nancy   Laverty, Chamindra   Phan, Han   Ghosh, Partha   Perlman, Seth   Scharf, Rebecca   mosher, Kathryn   Bernes, Saunder   emerson, Jane   Smith, Edward   Binks, Michael   Awadalla, Mark   Marban, Linda   

Session Name:LS2: Late-breaking Science 2  

Topic:Child Neurology and Developmental Neurology  

Program Number:Child Neurology and Developmental Neurology  

Author Institution:Department of Pediatrics, Dallas, TX  UC Davis Dept. of PM&R, Sacramento, CA  Arkansas Childrens Hospital/UAMS, Little Rock, AR  Washington University in St Louis, Saint Louis, MO  USC/Children'S Hospital of Los Angeles, Los Angeles, CA  Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL  University of Utah, Salt Lake City, UT  Consultant, WA  Capricor Therapeutics Inc, NC  Capricor Therapeutics Inc., UT  Children's Hospital Colorado, CO  Cincinnati Childrens Hospital Medical Center, Cincinnati, OH  University of Iowa - Dept of Pediatrics, Iowa City, IA  Children's Wisconsin, Milwaukee, WI  UC San Diego, San Diego, CA  Rare Disease Research, LLC, Atlanta, GA  Boston Children'S Hospital, Boston, MA  Seattle Children's Hospital, Seattle, WA  University of Virginia Children’s Hospital, VA  Akron Children's Hospital, OH  Phoenix Childrens Hospital, Phoenix, AZ  University of Missouri School of Medicine, Columbia, MO  Rare Disease Research - NC, LLC, Hillsborough, NC  Capricor Therapeutics Inc, Cambridge, MA  Capricor Therapeutics Inc, NJ  capricor Therapeutics Inc, CA  

Electrophysiological Improvements in Patients with Dravet Syndrome Following Treatment with Zorevunersen, an Investigational Antisense Oligonucleotide

Selective Potentiation of NaV1.1 Channels by Small Molecule XPC-837 in Dravet Mice Suppresses Spontaneous Seizures, Prevents SUDEP, and Increases Long Term Potentiation