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Nervenheilkunde 2023; 42(01/02): 80-86
DOI: 10.1055/a-1959-6579
Schwerpunkt

Aktuelle Therapien von Ataxien

Current therapies of ataxias
Andreas Thieme
1   Klinik für Neurologie und Zentrum für Translationale Neuro- und Verhaltenswissenschaften (C-TNBS), Universitätsmedizin Essen
,
Dagmar Timmann
1   Klinik für Neurologie und Zentrum für Translationale Neuro- und Verhaltenswissenschaften (C-TNBS), Universitätsmedizin Essen
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ZUSAMMENFASSUNG

Ataxien sind eine heterogene Gruppe von Erkrankungen. Sie können in jedem Lebensalter auftreten und haben verschiedene Ursachen. Die meisten Ataxien gehören zu den seltenen Erkrankungen und viele sind genetisch bedingt. Bislang beschränkt sich die Behandlung von Ataxien meist auf die symptomatische Therapie, insbesondere mittels Physiotherapie und Logopädie sowie auf die medikamentöse Behandlung von Begleitsymptomen. Eine wirksame „antiataktische“ medikamentöse Therapie steht unverändert nicht zur Verfügung. Nur für wenige Ataxien mit bekanntem Stoffwechseldefekt gibt es etablierte, zielgerichtete Therapieansätze. Tierexperimentell werden zunehmend genetische Therapien entwickelt. Erste klinische Sicherheitsstudien mit Antisense-Oligonukleotiden haben begonnen oder stehen kurz davor.

ABSTRACT

Ataxias are a heterogeneous group of disorders. They can occur at any age and have various etiologies. Most ataxias are rare diseases, and many have a genetic cause. So far, the treatment of ataxias has mostly been limited to symptomatic therapy, in particular physiotherapy and speech therapy as well as drug treatment of concomitant symptoms. Yet, there is no “anti-ataxic” drug. Established targeted drug treatments are only available for a few ataxias with known metabolic defects. Genetic therapies are increasingly being developed in animal experiments. First clinical safety studies with antisense oligonucleotides have started or are about to start.

Schlüsselwörter

Physiotherapie - Gentherapie - medikamentöse Therapie - symptomatische Therapie - zielgerichtete Therapie

Key words

Physiotherapy - gene therapy - drug therapy - symptomatic therapy - targeted therapy


Publication History

Article published online:
13 February 2023

© 2023. Thieme. All rights reserved.

© Georg Thieme Verlag KG Stuttgart · New York
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