Drug repurposing in Rett and Rett-like syndromes: a promising yet underrated opportunity?

Claudia Fuchs*, Peter A. C. 't Hoen, Annelieke R. Mueller, Friederike Ehrhart, Clara D. M. Van Karnebeek

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review

Abstract

Rett syndrome (RTT) and Rett-like syndromes [i.e., CDKL5 deficiency disorder (CDD) and FOXG1-syndrome] represent rare yet profoundly impactful neurodevelopmental disorders (NDDs). The severity and complexity of symptoms associated with these disorders, including cognitive impairment, motor dysfunction, seizures and other neurological features significantly affect the quality of life of patients and families. Despite ongoing research efforts to identify potential therapeutic targets and develop novel treatments, current therapeutic options remain limited. Here the potential of drug repurposing (DR) as a promising avenue for addressing the unmet medical needs of individuals with RTT and related disorders is explored. Leveraging existing drugs for new therapeutic purposes, DR presents an attractive strategy, particularly suited for neurological disorders given the complexities of the central nervous system (CNS) and the challenges in blood-brain barrier penetration. The current landscape of DR efforts in these syndromes is thoroughly examined, with partiuclar focus on shared molecular pathways and potential common drug targets across these conditions.
Original languageEnglish
Article number1425038
Number of pages9
JournalFrontiers in medicine
Volume11
DOIs
Publication statusPublished - 29 Jul 2024

Keywords

  • Rett syndrome
  • CDKL5 deficiency disorder
  • FOXG1-syndrome
  • shared molecular pathways
  • common drug targets
  • drug repurposing
  • TERM SERTRALINE TREATMENT
  • MOUSE MODEL
  • RECEPTOR AGONIST
  • DOUBLE-BLIND
  • MUTANT MICE
  • OPEN-LABEL
  • GANAXOLONE
  • BRAIN
  • DISEASE
  • MECP2

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