Fragile X Syndrome Market Spotlight Report 2021: 10-year Disease Incidence and Prevalence Forecasts, Probability of Success, Licensing and Asset Acquisition Deals, Drug-specific Revenue Forecasts – ResearchAndMarkets.com

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DUBLIN–(BUSINESS WIRE)–The “Market Spotlight: Fragile X Syndrome” report has been added to ResearchAndMarkets.com’s offering. Fragile X syndrome (FXS) is a genetic disorder leading to various developmental problems ranging from learning disabilities to cognitive impairment. FXS is caused by mutation of the fragile X mental retardation 1 (FMR1) gene, which usually makes a protein known as fragile X mental retardation protein (FMRP). FMRP is required for normal brain development, and individuals with FXS do not make this protein. Individuals who have other fragile X-associated disorders (FXTAS) have mutations in their FMR1 gene, but they generally make some FMRP. Key Takeaways Based on cohorts of children with special educational needs, the prevalence of fragile X syndrome (FXS) is estimated to be 1 in 4,000 in males, and 1 in 8,000 in females. The majority of industry-sponsored drugs in active clinical development for FXS are in Phase II, with two drugs in Phase III. Therapies in active clinical development for FXS focus on targets such as cannabinoid-1/cannabinoid-2 receptor, phosphodiesterase 4, GABA-A receptor, insulin-like growth factor-1 receptor, GABA-B receptor, 11-beta-hydroxylase, 11 betahydroxysteroid dehydrogenase type 1, and potassium channels. The majority of these pipeline drugs are administered via the oral route. The overall likelihood of approval of a Phase I neurodevelopmental disorders asset is 2.7%, and the average probability a drug advances from Phase III is 20%. Drugs, on average, take 12.4 years from Phase I to approval, compared to 10.0 years in the overall neurology space. The distribution of clinical trials across Phase I-IV indicates that the majority of trials for FXS have been in the early and midphases of development, with 77% of trials in Phase I-II, and only 23% in Phase III-IV. The US has a substantial lead in the number of FXS clinical trials globally. France and Spain lead the major…

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Source : Fragile X Syndrome Market Spotlight Report 2021: 10-year Disease Incidence and Prevalence Forecasts, Probability of Success, Licensing and Asset Acquisition Deals, Drug-specific Revenue Forecasts – ResearchAndMarkets.com

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