The medication patterns of spinocerebellar ataxia type 3 mutation carriers enrolled in the ESMI Cohort

Spinocerebellar ataxia type 3 (SCA3) is one of the most common dominantly inherited ataxias worldwide. Despite research advances, no approved disease-modifying treatment exists, and management focuses on symptom alleviation and functional capacity maximization. Symptomatic treatment guidelines are s...

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Main Authors: Silva, Patrick (Author) , Costa, Marina A. (Author) , Gaspar, Laetitia (Author) , Durães, João (Author) , Cunha, Inês (Author) , Ribeiro, Joana A. (Author) , Januário, Cristina (Author) , Oliveiros, Bárbara (Author) , Hübener-Schmid, Jeannette (Author) , Faber, Jennifer (Author) , Raposo, Mafalda (Author) , Lima, Manuela (Author) , Garcia-Moreno, Hector (Author) , Giunti, Paola (Author) , Beichert, Lukas David (Author) , Schöls, Ludger (Author) , van de Warrenburg, Bart P. (Author) , de Vries, Jeroen (Author) , Thieme, Andreas (Author) , Reetz, Kathrin (Author) , Jacobi, Heike (Author) , Infante, Jon (Author) , Klockgether, Thomas (Author) , Ferreira, Ana (Author) , Rosa, Ana (Author) , Gonzalez, Carlos (Author) , Gonzalez-Robles, Cristina (Author) , Timmann-Braun, Dagmar (Author) , Erdlenbruch, Friedrich (Author) , Lemos, João (Author) , Vasconcelos, João (Author) , Teves, Luís (Author) , Pires, Paula (Author) , Lopes, Pedro (Author) , Coelho, Pedro (Author) , Kay, Teresa (Author) , de Almeida, Luís Pereira (Author) , Santana, Magda M. (Author)
Format: Article (Journal)
Language:English
Published: 17 October 2025
In: CNS drugs
Year: 2026, Volume: 40, Issue: 2, Pages: 233-246
ISSN:1179-1934
DOI:10.1007/s40263-025-01237-w
Online Access:Verlag, kostenfrei, Volltext: https://doi.org/10.1007/s40263-025-01237-w
Verlag, kostenfrei, Volltext: https://link.springer.com/article/10.1007/s40263-025-01237-w?utm_source=getftr&utm_medium=getftr&utm_campaign=getftr_pilot&getft_integrator=clarivate
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Author Notes:Patrick Silva, Marina A. Costa, Laetitia Gaspar, João Durães, Inês Cunha, Joana A. Ribeiro, Cristina Januário, Bárbara Oliveiros, Jeannette Hübener-Schmid, Jennifer Faber, Mafalda Raposo, Manuela Lima, Hector Garcia-Moreno, Paola Giunti, Lukas Beichert, Ludger Schöls, Bart P. van de Warrenburg, Jeroen de Vries, Andreas Thieme, Kathrin Reetz, Heike Jacobi, Jon Infante, Thomas Klockgether, Ana Ferreira, Ana Rosa, Carlos Gonzalez, Cristina Gonzalez-Robles, Dagmar Timmann, Friedrich Erdlenbruch, João Lemos, João Vasconcelos, Luís Teves, Paula Pires, Pedro Lopes, Pedro Coelho, Teresa Kay, Luís Pereira de Almeida, Magda M. Santana, ESMI Study Group
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Summary:Spinocerebellar ataxia type 3 (SCA3) is one of the most common dominantly inherited ataxias worldwide. Despite research advances, no approved disease-modifying treatment exists, and management focuses on symptom alleviation and functional capacity maximization. Symptomatic treatment guidelines are scarce, leaving decisions to physicians’ discretion. The lack of studies on SCA3 symptom management hinders therapy standardization. The aim of this study was to investigate medication-usage patterns among SCA3 mutation carriers and controls included in the multicentric European Spinocerebellar Ataxia Type-3/Machado-Joseph Disease Initiative (ESMI) cohort.
Item Description:Gesehen am 06.03.2026
Physical Description:Online Resource
ISSN:1179-1934
DOI:10.1007/s40263-025-01237-w