Cystinosis Disease Therapeutics Market: Introduction
- Cystinosis is a rare genetic disorder caused by the accumulation of cystine in different organs such as pancreas, brain, kidneys, eyes, and muscles. Increase in cystine in the cells may damage the nervous tissue and also impact other organs. Prevalence of cystinosis is higher in children as compared to that in adults. Juvenile nephropathic cystinosis is considered to be highly common in children. Onset and symptoms of cystinosis disease varies differently in various patients and widely impact the kidneys and eyes in patients.
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- Patients suffering from nephropathic or intermediate cystinosis drive the urgent need for kidney transplant. Cystinosis is caused by the mutation of CTNS gene and is considered as autosomal recessive disease. Cystinosis is classified as lysosomal storage disorder and forms as crystal in cells and tissues, which starts damaging and affecting the organs.
- Cystinosis is fatal in younger age patients and development of cysteamine and advanced kidney transplantation offers improvement in the cystinosis disease treatment and increases the life expectancy of the patients beyond 50 years of age. Accurate diagnosis at the right time offers early detection and leads to the effective treatment of patients.
Key Drivers, Restraints, and Opportunities of Cystinosis Disease Therapeutics Market
- Increasing awareness about cystinosis disease and its accurate treatments is a major factor that is estimated to boost the cystinosis disease therapeutics market during the forecast period. Apprehension of mutation screening as a treatment, in order to avoid gene mutation, is likely to boosts the global cystinosis disease therapeutics market during the forecast period. Continuous research & clinical trials for the treatment of cystinosis disease and development of potential treatment are also key factors that are estimated to propel the cystinosis disease therapeutics market during the forecast period.
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- Rise in number of cases of kidney damage, known as renal Fanconi syndrome, and poor growth and development of infants drive the need of accurate treatment for cystinosis disease. Underdiagnoses of this disease restrains the development of therapeutics for the treatment of cystinosis disease.
- Rise in cases of cystinosis in infants and increase in incidences in adults is another factor that is estimated to fuel the global market during the forecast period. For instance, cystinosis affects approx. 1 in 100,000 to 200,000 infants globally, and the prevalence of cystinosis disease is higher in France, thereby affecting around 1 in 26,000 individuals in the country. Increase in standard diagnostic tools is another factor that is estimated to propel the market during the forecast period.
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North America to Dominate Global Cystinosis Disease Therapeutics Market
- North America dominated the global cystinosis disease therapeutics market in 2024, followed by Europe and Asia Pacific. Increasing patient burden and presence of companies offering treatment of the cystinosis disease are likely to propel the global market during the forecast period. As per research, cystinosis disease affects 2,000 people across globe and 500 people are estimated to be affected by the disease in North America every year. This is likely to drive the cystinosis disease therapeutics market in North America during the forecast period. Technological advancements, innovations in the development of products, and receiving FDA approval for various products portfolio are other factors that are estimated to fuel the global cystinosis disease therapeutics market during the forecast period.
- Prevalence of cystinosis disease is higher in Europe, such as in France and Germany, which is expected to drive the need for treatment of the condition. Moreover, awareness about cysteamine for treatment and preference of kidney transplant by experts for growing patient burden are other factors boosting the cystinosis disease therapeutics market in the region. Rise in renal Fanconi syndrome in children due to cystinosis disease is also another factor that is projected to drive the market during the forecast period. Incidence rate of cystinosis disease were reported to be 1 per 167,000 in France, 1 per 192,000 in Australia, 1 per 179,000 in Denmark, 1 per 115,000 in Denmark in 2017. All these statistics are likely to boost the cystinosis disease therapeutics market in Europe.
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Key Companies in Global Cystinosis Disease Therapeutics Market
- AVROBIO, Inc.
- BTL
- Chiesi Farmaceutici SpA
- Horizon Therapeutics plc
- Mylan N.V.
- Eloxx Pharmaceuticals
- Leadiant Biosciences, Inc.
- Recordati Rare Diseases
- GlaxoSmithKline plc.
- Novartis AG
- Gilead Sciences
- Sigma-Aldrich
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