Abstract: The present invention relates to compounds or pharmaceutical compositions for treating neurological disorders. In particular, the invention relates to a modulator of gamma-Aminobutyric acid (GABA) signaling for use in treating a neurological disorder in a subject with Cyfip1 haploinsufficiency. Furthermore, the present invention relates to a modulator of Aralar for use in treating a neurological disorder. The invention further relates to non-medical uses of the compounds or compositions of the invention. Furthermore, the invention relates to a method for identifying a modulator of Aralar and/or a neuroactive drug.

Abstract: The present application relates to Fragile X syndrome and the treatment thereof. It was found that ADAM10 (A Dis-integrin And Metalloproteinase domain 10), the most likely candidate for ?-secretase, involved in proteolytic cleavage of APP at the plasma membrane, was upregulated in Fmr1 KO mice, a model for Fragile X disease. Moreover, it could be shown that reducing ADAM10 activityin vitro and in vivo, improves the Fragile X phenotype, i.a. by rescuing spine dysmorphogenesis and exaggerated mGluR-dependent LTD.

Abstract: The present application relates to the field of cancer. Surprisingly, it was shown that the Fragile X mental retardation gene (Fmr1) products such as the FMRP protein, primarily implicated in mental retardation, are upregulated in metastasizing tumours. It is shown how Fmr1 gene products can be used as a marker for cancer metastasis and how inhibition of these gene products may help prevent or reduce metastasis.