080210xf's Blog

L'X fragile sera vaincu | Fragile X will be conquered

Fragile X Therapeutics Programs Reviewed by NeuroInvestment

CARDIFF, CA–(Marketwire – December 1, 2010) – NI Research has released the December issue of NeuroInvestment, which features a comprehensive review of therapeutics for the treatment of Fragile X.

Fragile X has become a focus for a growing number of pharmaceutical companies as they have considered the potential value of addressing an orphan disorder which is a significant neurodevelopmental disorder in its own right, the single most common form of inherited mental disability/retardation. Successful disease-modification in Fragile X may also be the gateway to disease-modifying treatments for autism, and perhaps even schizophrenia.


The Fragile X mutation leads to the underproduction of FMRP, an essential mediator of synaptic protein synthesis, leading to defects in both structure and cognitive function. The major approaches to Fragile X, beyond the crude attempts at neuroleptic symptom control currently available, include mGluR5 antagonism (Seaside Therapeutics, Novartis, Roche), GABA-B antagonism (Seaside Therapeutics), and PAK inhibition (Afraxis). Other potential strategies include neurotrophic/protective drugs (Neuren, Cortex Pharmaceuticals) and oxytocin (Cypress Bioscience). Hint of concept has been established in pilot studies conducted by Novartis and Seaside Therapeutics.


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