Fragiele-X-syndroom: nieuwe therapeutische strategieën

Translated title of the contribution: Fragile X syndrome: new therapeutic strategies: new therapeutic strategies

S Zeidler, B Dierckx, K Lubbers, A M van Eeghen, C R Lincke, J A Kievit, R Willemsen, A Rietman

Research output: Contribution to journalReview articleProfessional


BACKGROUND: Fragile X syndrome (fxs) is the most common hereditary cause of intellectual disability and autism spectrum disorders. Targeted treatment is currently lacking. In the past decades an enormous amount of knowledge has been obtained concerning the involved molecular pathways, introducing potential targets for disease modifying therapy.<br/> AIM: To present an overview of the development of targeted treatment for fxs.<br/> METHOD: Several important publications were collected and indexed.<br/> RESULTS: While preclinical animal model studies with targeted interventions are promising, the translation to the clinic has been disappointing.<br/> CONCLUSION: Targeted treatment for fxs is necessary and could be applied in other causes of autism spectrum disorders and intellectual disability. Factors relating to translation, study design and outcome measures are possibly contributing to the disappointing results. The clustering of patient care in a center of expertise is required to clinically implement future therapeutic strategies and to facilitate research. In addition, this improves patient care, one example being the recent medical guideline for children with fxs.

Translated title of the contributionFragile X syndrome: new therapeutic strategies: new therapeutic strategies
Original languageDutch
Pages (from-to)338-342
Number of pages5
JournalTijdschrift voor Psychiatrie
Issue number5
Publication statusPublished - 17 May 2018


  • Animals
  • Autism Spectrum Disorder
  • Child
  • Clinical Trials as Topic
  • Disease Models, Animal
  • Fragile X Syndrome/genetics
  • Humans
  • Intellectual Disability
  • Molecular Targeted Therapy

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