Fragile X Syndrome Market Size and Trend Report including Epidemiology and Pipeline Analysis, Competitor Assessment, Unmet Needs, Clinical Trial Strategies and Forecast, 2021 - 2030
Summary
Fragile X syndrome (FXS) (ICD-10: Q99.2) is an X-linked genetic condition and is the most common cause of inherited learning disability. FXS is caused by expansion of a cytosine, guanine, and guanine (CGG) trinucleotide repeats in the 5’ untranslated region of the fragile X mental retardation 1 (FMR1) gene. This expansion results in transcriptional silencing of the FMR1 gene with a subsequent reduction or absence of fragile X mental retardation protein (FMRP) expression. FMRP is an important mediator in dendritic maturity and synaptic plasticity.
FXS is a lifelong disorder associated with a spectrum of developmental and behavioral issues, including psychiatric symptoms (anxiety, attention deficit hyperactivity disorder [ADHD], and poor eye contact) and neurologic symptoms (seizures and movement disorders). Other common features of the syndrome include characteristic physical attributes (large and prominent ears, long face, and flat feet) as well as deafness, due to the high recurrence of ear infections. The severity of these symptoms is dependent on the amount of FMRP protein that is produced, which in turn is dependent on both the number of CGG repeats as well as the degree of methylation in the FMR1 gene. As females have two X chromosomes and thus two copies of the FMR1 gene, females often have milder symptoms than males.
The two clinically and commercially relevant segments considered for the classification of FXS in this report are segmented into three groups by age: pediatric patients aged 0-11 years, adolescent patients aged 12-17, and adult patients aged 18+.
This report covers 2MM (US and Germany) and provides an Excel-based forecast model for the Fragile X Syndrome market through 2030.
Key Highlights
Companies Mentioned
Acadia Pharmaceuticals
Advanz Pharma Corp
Boehringer Ingelheim
Confluence Pharmaceuticals
Eli Lilly
GlaxoSmithKline
H. Lundbeck
Janssen
Neuren Pharmaceuticals
Novartis
Otsuka Pharmaceuticals<
Learn how to effectively navigate the market research process to help guide your organization on the journey to success.
Download eBook