Blueprint for clinical N-of-1 strategies with off-label precision treatments in monogenic epilepsies

Blueprint for clinical N-of-1 strategies with off-label precision treatments in monogenic epilepsies

Abstract Precision treatments for monogenic epilepsies, i.e. treatments that can at least partially reverse the biochemical consequences of a pathogenic gene variant, have been gradually emerging over the years. To date, however, information on the efficacy of these treatments is mostly based on cas...

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Main Authors: Victoria M. Defelippe, Eva H. Brilstra, Willem M. Otte, Ghislaine J. M. W. van Thiel, Helen J. Cross, Finbar O’Callaghan, Valentina De Giorgis, Emilio Perucca, Kees P. J. Braun, Floor E. Jansen
Format: Article
Language:English
Published: BMC 2025-06-01
Series:Orphanet Journal of Rare Diseases
Subjects:
N-of-1 trial
Single case-studies
Precision medicine
Quality improvement of care
Epilepsy
Online Access:https://doi.org/10.1186/s13023-025-03750-z
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https://doi.org/10.1186/s13023-025-03750-z

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