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PK/PD Modeling to Inform Clinical Development of an Adeno-associated Virus Gene Transfer Therapy for Duchenne Muscular Dystrophy

Author:East, Lilly   Potter, Rachael   Snedeker, John   Haile, Alex   Wier, Chris   Rodino-Klapac, Louise   

Session Name:S48: Therapeutics for Muscle Disease  

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:S48.005  

Author Institution:Sarepta Therapeutics, Inc. , Cambridge, MA  Sarepta Therapeutics, Inc., Cambridge, MA  

Evaluating Pharmacology and Efficacy of Delandistrogene Moxeparvovec in Young and Aged DMDmdx Rats

Author:Potter, Rachael   Wier, Chris   Cooper-Olson, Grace   Wheeler, Esther   Anderbery, Emily   Kempton, Amber   Clements, Leah   Adegboye, Kaitlin   Haile, Alex   Peterson, Ellyn   Rodino-Klapac, Louise   

Session Name:P3: Neuromuscular Disease: Inherited Muscle Disorders 1  

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:P3.008  

Author Institution:Sarepta Therapeutics, Inc. , Cambridge, MA  Sarepta Therapeutics, Inc., Cambridge, MA