DelveInsight’s “Fragile X Syndrome Market Insights, Epidemiology, and Market Forecast-2034″ report offers an in-depth understanding of the Fragile X Syndrome, historical and forecasted epidemiology as well as the Fragile X Syndrome market trends in the United States, EU4 (Germany, Spain, Italy, France, and United Kingdom) and Japan.
To Know in detail about the Fragile X Syndrome market outlook, drug uptake, treatment scenario and epidemiology trends, Click here; Fragile X Syndrome Market Forecast
Some of the key facts of the Fragile X Syndrome Market Report:
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The Fragile X Syndrome market size is anticipated to grow with a significant CAGR during the study period (2020-2034)
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In November 2024, Chris Peetz, the CEO of Mirum, highlighted the company’s strong performance in the third quarter, noting growth across all three commercial medicines, particularly the acceleration of LIVMARLI PFIC sales. He also emphasized significant progress in the clinical pipeline, including the granting of breakthrough designation for volixibat in PBC and the initiation of the EXPAND study for LIVMARLI in other cholestatic pruritus indications. Additionally, the rare genetic neurology franchise is advancing, with a December 28 PDUFA date for chenodiol for CTX and the addition of Phase 2-ready MRM-3379 for Fragile X syndrome.
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In October 2024, Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY) will present an extensive update on its pipeline during its Investor Day event today, highlighting new data from its orexin-2 receptor agonist and next-generation pitolisant programs, along with long-term extension data from its pitolisant program for idiopathic hypersomnia. The company will also share updates on its strategic acquisitions in Fragile X syndrome (FXS) and rare epilepsies, which have bolstered its late-stage development pipeline.
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In May 2024, Spinogenix, Inc., a clinical-stage biopharmaceutical company focused on developing first-in-class therapies to restore synapses and enhance the lives of patients globally, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to SPG601 for the treatment of Fragile X Syndrome (FXS).
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In April 2024, Spinogenix received approval from the U.S. Food and Drug Administration (FDA) to conduct a Phase II clinical trial of SPG601 for Fragile X Syndrome (FXS). This approval coincides with the European Commission granting orphan drug status to Shionogi’s zatolmilast (BPN14770).
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DelveInsight's epidemiology model estimates that the total diagnosed prevalent cases of FXS in the 7MM were around 122,000 in 2023. This figure is expected to grow during the forecast period (2024-2034) due to heightened awareness, improved screening, and advancements in genetic testing.
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In 2023, the US had the highest number of diagnosed prevalent cases of FXS, totaling around 71,000 cases, whereas Spain reported the lowest, with nearly 5,900 cases.
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In 2023, among the EU4 and the UK, Germany had the highest number of diagnosed prevalent cases of FXS, with about 9,700 cases, followed by France with around 7,900 cases and the UK with nearly 7,800 cases.
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In 2023, around 42,000 males and 29,000 females in the US were diagnosed with FXS, indicating a greater prevalence in males than in females.
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In Japan, in 2023, there were about 5,700 cases in the 0–11 age group, 2,300 cases in the 12–17 age group, and 5,000 cases among individuals over 18, reflecting differences in prevalence across age groups.
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Key Fragile X Syndrome Companies: Zynerba Pharmaceuticals, Confluence Pharmaceuticals, Tetra Therapeutics, Neuren Pharmaceuticals, Healx, Craig Erickson, Nova Mentis Life Science, and others
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Key Fragile X Syndrome Therapies: Zygel, Acamprosate, BPN14770, Trofinetide, Sulindac (HLX-0201), Gaboxadol, ZYN002, Psilocybin, and others
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The Fragile X Syndrome epidemiology based on gender analyzed that males are affected more as compared to females, in the case of Fragile X Syndrome (FXS)
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The Fragile X Syndrome market is expected to surge due to the disease's increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Fragile X Syndrome pipeline products will significantly revolutionize the Fragile X Syndrome market dynamics.
Fragile X Syndrome Overview
Fragile X Syndrome (FXS) is a genetic disorder caused by a mutation in the FMR1 gene on the X chromosome. It is the most common inherited cause of intellectual disability and a leading genetic cause of autism. FXS primarily affects males, but females can also be impacted, though often less severely. Symptoms include developmental delays, learning difficulties, behavioral challenges, anxiety, and physical features like a long face, prominent ears, and flexible joints. There is no cure, but early interventions such as therapy, educational support, and medications can help manage symptoms and improve quality of life.
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Fragile X Syndrome Epidemiology
The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2020 to 2034. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends.
Fragile X Syndrome Epidemiology Segmentation:
The Fragile X Syndrome market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into:
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Total Prevalence of Fragile X Syndrome
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Prevalent Cases of Fragile X Syndrome by severity
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Gender-specific Prevalence of Fragile X Syndrome
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Diagnosed Cases of Episodic and Chronic Fragile X Syndrome
Download the report to understand which factors are driving Fragile X Syndrome epidemiology trends @ Fragile X Syndrome Epidemiology Forecast
Fragile X Syndrome Drugs Uptake and Pipeline Development Activities
The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Fragile X Syndrome market or expected to get launched during the study period. The analysis covers Fragile X Syndrome market uptake by drugs, patient uptake by therapies, and sales of each drug.
Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.
The report also covers the Fragile X Syndrome Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.
Fragile X Syndrome Therapies and Key Companies
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Zygel: Zynerba Pharmaceuticals
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Acamprosate: Confluence Pharmaceuticals
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BPN14770: Tetra Therapeutics
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Trofinetide: Neuren Pharmaceuticals
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Sulindac (HLX-0201): Healx
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Gaboxadol: Craig Erickson
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ZYN002: Zynerba Pharmaceuticals
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Psilocybin: Nova Mentis Life Science
Discover more about therapies set to grab major Fragile X Syndrome market share @ Fragile X Syndrome Treatment Market
Fragile X Syndrome Market Strengths
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Growing Research and development are increasing the demand for FXS treatment.
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Advances in DNA technology, including CRISPRCas9, have been used to correct the expanded CGG repeat in induced pluripotent stem cells. Eventually, it might be possible to add these corrected stem cells into the central nervous systems of those with FXS
Fragile X Syndrome Market Opportunities
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The rise in the prevalence of FXS stimulates the research and development of the drug, as it is likely to provide an appropriate environment for newer products to be profitable.
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Increasing population gives a lucrative opportunity to increase the awareness and innovation of therapeutics to drive FXS market.
Scope of the Fragile X Syndrome Market Report
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Study Period: 2020–2034
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Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]
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Key Fragile X Syndrome Companies: Zynerba Pharmaceuticals, Confluence Pharmaceuticals, Tetra Therapeutics, Neuren Pharmaceuticals, Healx, Craig Erickson, Nova Mentis Life Science, and others
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Key Fragile X Syndrome Therapies: Zygel, Acamprosate, BPN14770, Trofinetide, Sulindac (HLX-0201), Gaboxadol, ZYN002, Psilocybin, and others
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Fragile X Syndrome Therapeutic Assessment: Fragile X Syndrome current marketed and Fragile X Syndrome emerging therapies
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Fragile X Syndrome Market Dynamics: Fragile X Syndrome market drivers and Fragile X Syndrome market barriers
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Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies
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Fragile X Syndrome Unmet Needs, KOL’s views, Analyst’s views, Fragile X Syndrome Market Access and Reimbursement
To know more about Fragile X Syndrome companies working in the treatment market, visit @ Fragile X Syndrome Clinical Trials and Therapeutic Assessment
Table of Contents
1. Fragile X Syndrome Market Report Introduction
2. Executive Summary for Fragile X Syndrome
3. SWOT analysis of Fragile X Syndrome
4. Fragile X Syndrome Patient Share (%) Overview at a Glance
5. Fragile X Syndrome Market Overview at a Glance
6. Fragile X Syndrome Disease Background and Overview
7. Fragile X Syndrome Epidemiology and Patient Population
8. Country-Specific Patient Population of Fragile X Syndrome
9. Fragile X Syndrome Current Treatment and Medical Practices
10. Fragile X Syndrome Unmet Needs
11. Fragile X Syndrome Emerging Therapies
12. Fragile X Syndrome Market Outlook
13. Country-Wise Fragile X Syndrome Market Analysis (2020–2034)
14. Fragile X Syndrome Market Access and Reimbursement of Therapies
15. Fragile X Syndrome Market Drivers
16. Fragile X Syndrome Market Barriers
17. Fragile X Syndrome Appendix
18. Fragile X Syndrome Report Methodology
19. DelveInsight Capabilities
20. Disclaimer
21. About DelveInsight
About DelveInsight
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It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate the business growth and overcome challenges with a practical approach.
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