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Eladocagene Exuparvovec Gene Therapy Improves Motor Development in Patients With Aromatic L-Amino Acid Decarboxylase Deficiency

Author:Russell, Alexis   Roubertie, Agathe   Chien, Yin-Hsiu   Lee, Ni-Chung   Sierra, Rafael   Turna, Jasmine   Wang, Antonia   Tai, Chun-Hwei   Hwu, Paul W-L   

Session Name:S34: Child Neurology and Developmental Neurology 2  

Topic:Child Neurology and Developmental Neurology  

Program Number:S34.004  

Author Institution:PTC Therapeutics, Inc., South Plainfield, NJ  University Hospital of Montpellier, Montpellier, France, Montpelier, France  National Taiwan University Hospital, Taipei, Taiwan  

Evaluation of the Biodistribution, Efficacy, and Side-Effect Profile of Deflazacort, Prednisolone and Vamorolone in a DMD Mouse Model

Author:Liu, Grace   Lipari, Phil   Mollin, Anna   Jung, Stephen   Teplova, Irina   Ma, Marcia   Li, Wencheng   Ying, Lanqing   Yeh, Shirley   Narasimhan, Jana   Trifillis, Panayiota   Leonardi, Efthimia   Welch, Ellen   Voronin, Gregory   Dong, Elaine   Mastrandrea, Nicholas   Blaize, Jonathan   Weetall, Marla   

Session Name:S34: Child Neurology and Developmental Neurology 2  

Topic:Child Neurology and Developmental Neurology  

Program Number:S34.010  

Author Institution:PTC Therapeutics, Inc., South Plainfield, NJ