Cystinosis Treatment Market Comprehensive study with key drivers: Horizon Pharma Plc., Novartis AG, Mylan N.V. and Avrobio, Inc., among others.
Cystinosis is a disorder which is characterized by the accumulation of cystine within cells. High quantity of cystine damages cells that form crystals, damaging organs and tissues. The kidney and eye are mostly susceptible to damage. However, thyroid, testes, muscles and pancreas can also be affected. Cystinosis is categorized into three types such as intermediate cystinosis, non-nephropathic or ocular, and nephropathic. Intermediate cystinosis is characterized by growth failure and Renal Fanconi syndrome but these symptoms are not notable at age ranging from 8 to 20 years. Non-nephropathic cystinosis commonly affects adults of middle age with an eye disorder and kidney disease. People with this disorder develop photophobia because of cystine crystal accumulation in eyes. Nephropathic cystinosis is categorized by symptoms similar to that of intermediate cystinosis.
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Major players in the market are Horizon Pharma Plc., Novartis AG, Mylan N.V. and Avrobio, Inc., among others.
Cystinosis Research Foundation, in 2016, rewarded $2.79 million in research in five countries, the funding for 17 scientific studies on pathology treatment and also potential cures for the disorder. It has helped to cure this rare disease. The Foundation announced that they might get FDA approval this year to advance the autologous stem cell treatment for clinical trials and for improvement of nanowafer treatment for the corneal cystinosis.
Business Intelligence: FDA approved delayed-release formulation for every twelve hour of cysteamine bitartrate for nephropathic cystinosis in pediatric and adults patients. FDA approval in 2015 was expanded from 6 years of age to 2 years of age. This reduces the burden from 6 hours to 12 hours daily. Most commonly known mutation linked with the cystinosis in Northern Europeans is 57-kb deletion, which involves first of ten exons. Avrobio is clinical stage Biotechnology Company, which is developing transformation in treatment for rare diseases. On October 4, 2017, it announced expansion of the pipeline to cystinosis.. Lysosomal storage disorders (LSDs) became the company’s fourth gene therapy. The program for cystinosis was licensed from the GenStem Therapeutics.
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Government funding to support treatment and diagnosis of cystinosis disorder is driving the global cystinosis market. FDA approved formulation of cysteamine bitartrate for 6 years and above patients and it was further expanded to include children aged from two years in 2015. Cysteamine reduces cystine levels in the cells it is a cystine-depleting agent. It has to be effective in delaying and preventing renal failure and also improving the growth of children with cystinosis disorder. FDA approved Cystaran in 2013 for treatment of the corneal cystine crystal accumulation. As soon as cystine crystals are found with corneas treatment with the cystaran should be initiated. The global cystinosis treatment market is expected to grow in the next few years because of the moderate availability of treatment options. On the other hand, less focus on research and development because of low prevalence rate and also less number of treatment option can restrain the cystinosis treatment market.
Global Composite Testing Market Segmentation:
• Renal transplantation
• Symptomatic Therapy
• Cysteine Depleting Therapy
By end user
• Specialty Clinics
• North America (U.S and Canada and rest of North America)
• Europe (Germany, France, Italy and Rest of Europe)
• Asia-Pacific (China, Japan, India, South Korea and Rest of Asia-Pacific)
• LAMEA (Brazil, Turkey, Saudi Arabia, South Africa and Rest of LAMEA)
Regional Analysis: Based on the regional analysis, the global skin care device devices market is segmented into North America, Europe, Asia-Pacific, and LAMEA. Rising awareness among patients for different treatment options available and number of increasing demand for the treatment will dominate the market in Europe. Also, due to increasing population, rising demand for the rapid diagnosis and increasing population.
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