Global Fragile X Syndrome (Fxs) Treatment Market - 2022-2029
The global fragile x syndrome (fxs) treatment market size was valued at US$ XX million in 2021 and is estimated to reach US$ XX million by 2029, growing at a CAGR of XX % during the forecast period (2022-2029).
FXS (Fragile X Syndrome) is a genetic condition. FXS is caused by mutations in the FMR1 gene, which scientists first discovered. The FMR1 gene usually produces the FMRP protein. FMRP is required for the development of the brain. People with FXS do not produce this protein.
The factors influencing the global fragile x syndrome (fxs) treatment market are the increasing research for effective treatment and the growing prevalence of fragile x syndrome (fxs).
The increasing research for effective treatment is expected to drive the market growth in the forecast period
FXS (Fragile X Syndrome) is a genetic condition. FXS is caused by mutations in the FMR1 gene, which scientists first discovered. The FMR1 gene usually produces the FMRP protein. FMRP is required for the development of the brain. People with FXS do not produce this protein. Other fragile X-associated disorders cause changes in the FMR1 gene, but most people produce some of the protein. Fragile X and its associated conditions, such as Fragile X-associated Primary Ovarian Insufficiency (FXPOI) and Fragile X-Associated Tremor/Ataxia Syndrome, as well as their diagnosis, treatment, management, prevention, and inheritance, are funded and researched by the National Institute of Child Health and Human Development (NICHD) (FXTAS). All three conditions are caused by changes in the same gene on the X chromosome.
Moreover, the Fragile X Research Foundation is working to find specific treatments and, eventually, a cure, but there is currently no cure. Fragile X syndrome affects most males and many females throughout their lives. The cost of treatment, special education, and lost income to society are enormous. The need for treatment-oriented research is critical.
Limited treatment options are expected to hamper the market growth
Although there is no cure for Fragile X syndrome, there are treatments that can help to alleviate its symptoms. Individuals with Fragile X who receive the proper education, therapy, and medications have the best chance of using all of their abilities and skills. Due to the limited treatment options, market growth is expected to be hampered.
The COVID-19 pandemic significantly impacted health systems' ability to continue providing essential health services. While increasing demand for COVID-19 care is putting pressure on health systems worldwide, it is critical to maintain preventive and curative services, especially for the most vulnerable populations, such as children, the elderly, people with chronic illnesses, minorities, and people with disabilities.
Moreover, infection with SARS-CoV-2 causes the release of inflammatory cytokines, which can cause an overabundance of inflammatory cytokines, leading to a cytokine storm and a worsening prognosis. Several signaling pathways, including the PI3K/Akt/mTOR and Ras/MAPK/ERK cascades, GSK-3 kinase, and NF-kB, are involved in these processes. In fragile X syndrome (FXS), the most common cause of inherited mental retardation and the most common genetic form of autism spectrum disorder, those signaling pathways, as well as IGF-1, are upregulated, leading to an increase in metalloproteinase 9 (MMP-9) levels, tissue damage, a pro-inflammatory state, and disruption of the blood-brain barrier. FXS and COVID-19 have so many links that suggest that they share pathophysiological mechanisms. FXS would be a significant risk factor for developing a severe form of COVID-19, and SARS-CoV-2 could exacerbate the symptoms and prognosis of both FXS patients and asymptomatic Fmr1 premutation carriers. This is expected to rise demand for the Fragile X Syndrome (Fxs) treatment. Hence, covid-19 is expected to have an impact on market growth.
Fragile X-Associated Tremor/Ataxia Syndrome (FXTAS) segment is expected to dominate the market growth
FXTAS (Fragile X-associated tremor/ataxia syndrome) is characterized by movement and thinking difficulties (cognition). FXTAS is a late-onset disorder that usually manifests after 50, and the signs and symptoms worsen as the patient gets older. Males are affected more frequently and severely than females by this condition. Damage to the cerebellum, the part of the brain that controls movement, and white matter, a type of brain tissue, can be seen with magnetic resonance imaging in those who are affected (MRI). FXTAS is characterized by movement problems and other impairments resulting from this damage.
Some people with FXTAS have issues with the autonomic nervous system, which controls involuntary body functions, resulting in bladder or bowel control issues. Cognitive impairments are common in people with FXTAS. They may experience short-term memory loss and executive function impairment, which is the ability to plan and carry out actions and develop problem-solving strategies. Impulse control, self-monitoring, appropriately focusing attention, and cognitive flexibility are all harmed when this function is lost. Anxiety, depression, moodiness, and irritability are common symptoms of FXTAS.
North America region is expected to hold the largest market share in the global fragile x syndrome (Fxs) treatment market
The growing prevalence of fragile x syndrome (Fxs) and better R&D in this region is expected to drive the market growth.
In the United States, studies estimate that 1 in 148 to 1 in 291 females and 1 in 290 to 1 in 855 males have a fragile X premutation. Moreover, fragile X syndrome affects about 1 in 2500-4000 males and 1 in 7000-8000 females. Female carrier status is estimated to affect one in every 130-250 people, while male carrier status affects one in every 250-800 people. Hence, with the growing prevalence of the disease in this region the market is expected to grow.
With mergers, acquisitions, and product launches, the global fragile x syndrome (Fxs) treatment market is moderately competitive. Some of the key players in the market are Ovid Therapeutics., Tetra Therapeutics, Neuren Pharmaceuticals, Anavex Life Sciences Corp., Confluence Pharmaceuticals, Zynerba Pharmaceuticals, Inc.
Overview: Ovid Therapeutics is a biotech company based in New York that develops medicines for people with rare neurological disorders using its BoldMedicine approach.
Product Portfolio: The company's OV101 (gaboxadol) is in clinical studies used in males aged 13 to 22 to treat Fragile X syndrome. A three-arm, double-blind clinical trial is evaluating the safety and tolerability of OV101 in Fragile X syndrome-affected adolescent and young adult males aged 13 to 22 years old.
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