Neuropediatrics 2025; 56(S 01): S1-S24
DOI: 10.1055/s-0045-1812170
Trofinetide for the Treatment of Rett Syndrome: Long-Term Safety and Efficacy Results
from the Open-Label LILAC and LILAC-2 Studies
Authors
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A. Huber
1
Acadia Pharmaceuticals GmbH, Medical Affairs, Basel, Switzerland
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A. Percy
2
University of Alabama at Birmingham, Birmingham, Alabama, United States
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J. Neul
3
Vanderbilt Kennedy Center, Nashville, Tennessee, United States
-
T. Benke
4
Children's Hospital of Colorado, Aurora, Colorado, United States
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E. Berry-Kravis
5
Rush University Medical Center, Chicago, Illinois, United States
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D. Glaze
6
Texas Children's Hospital, Houston, United States
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E. Marsh
7
Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
-
K. Bishop
8
Acadia Pharmaceuticals, San Diego, California, United States
-
J. Youakim
8
Acadia Pharmaceuticals, San Diego, California, United States
