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Fragile-x Syndrome Therapeutics Pipeline Analysis 2017 - Designation, Collaboration, and Other Developments


Due to lack of awareness among people regarding genetic disorders which leads to mutations in one’s DNA, the prevalence of fragile-x syndrome is increasing, across the globe. Smoking, alcohol consumption during pregnancy and hereditary disorders are few of the primary reasons to for the increase in prevalence of fragile-x syndrome. The therapeutic pipeline of fragile-x syndrome is expected to grow more rapidly in the future on account of active involvement of pharmaceuticals & biotechnology companies carrying out research programs to develop effective therapeutic agents or drugs for eradicating fragile-x syndrome. According to a study conducted by Centers for Disease Control and Prevention (CDC) in 2012, around one female in every 151 females & one male in every 468 males in the U.S., was affected by fragile-x syndrome. Moreover, according to the study, occurrence of fragile-x syndrome is higher in females as compared to males.  

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Fragile-x syndrome is a genetic disorder that affects the development and learning capabilities of an individual. It is also called as Marker-x syndrome and Martin-Bell syndrome. Fragile-x syndrome causes trinucleotide repeat in Fragile-x mental retardation 1(FMR-1) gene present on X chromosome. This gene is responsible for making a protein called Fragile-x mental retardation protein (FMRP), which is required for normal neural development. In case of an individual suffering from fragile-x syndrome, production of FMRP is constrained due to the repetition of CGG (trinucleotide) in FMR1 gene. The symptoms associated with fragile-x syndrome are mental retardation, learning disability, impulsiveness, stuttering, development delays, seizures, depression, hyperactivity, etc.

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Some of the companies having a pipeline of Fragile-x syndrome therapeutics include Ovid therapeutics, Marinus pharmaceuticals, Neuren pharmaceuticals, Bellus health Inc., AMO pharmaceuticals, Eli-lly& co., Anavex Life Sciences Corp., Kareus Therapeutics, and Zynerba Pharmaceuticals, Inc.