Cystinosis Treatment Market Growth Opportunities With Top Companies Avrobio, Inc., Gilead, Mylan N.V., Novartis Ag, And Orizon Pharma Plc

Cystinosis is a condition characterized by accumulation of the cystine (amino acid) within cells. High amount of cystine damages cells and often forms crystals that can damage tissues and organs. The eyes and kidneys are mainly susceptible to damage; however, the pancreas, muscles, testes, and thyroid can also be affected. Cystinosis is categorized into three different types; nephropathic, non-nephropathic or ocular, and intermediate cystinosis. Nephropathic (infantile) cystinosis is the most severe and common form of cystinosis. Renal Fanconi syndrome and growth failure are usually the first noticeable symptoms of this disorder.

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Cysteine depleting therapy can significantly reduce cysteine levels inside the cells. It improves growth and decreases kidney damage in children. Symptomatic therapy treats renal Fanconi syndrome with a high intake of electrolytes and fluids to prevent extreme body dehydration. Sodium bicarbonate, potassium, sodium citrate, and magnesium can help to keep standard electrolyte stability. Acetyl cholinesterase (ACE) inhibitors are also used sometimes to limit the growth of the renal disease.

"Cystinosis Treatment Market Dynamics"

Various government grants to support the diagnosis and treatment of cystinosis is boosting growth of the market. For instance, in 2013, the Cystinosis Research Foundation awarded six grants worth US $1.29 million to Cystinosis researchers for developing better treatments and a cure for the disease. Organizations such as Cystinosis Research Network are responsible to spread awareness regarding disease by celebrating Cystinosis Awareness Day on May 7, every year. Furthermore, product approval by the Food and Drug Administration (FDA) is expected to boost growth of the cystinosis treatment market over the forecast period. In 2013, the FDA approved the delayed-release formulation of cysteamine bitartrate (DR-C) for patients aged 6 years and above, and the label was expanded in 2015 to include children age two years.

Cysteamine is a cystine-depleting agents that reduces cystine levels in cells. It has proven to be effective in preventing or delaying renal failure and improving growth of children with Cystinosis. In 2013, FDA approved Cystaran (cysteamine ophthalmic solution) for the treatment of corneal cystine crystal accumulation. Treatment with Cystaran should be initiated as soon as cystine crystals are found within the corneas. Lack of focus on R&D due to low prevalence rate and limited treatment options can negatively impact cystinosis treatment.

"Cystinosis Treatment Market Competitive Landscape"

Key players operating in the cystinosis treatment market include Avrobio, Inc., Gilead, Mylan N.V., Novartis AG, and Orizon Pharma Plc. Companies in the cystinosis treatment market are focusing on new product development to gain major revenue share. For instance, Avrobio Inc. is planing to initiate clinical development of two gene therapy candidates for cystinosis and Gaucher disease by mid-2019.

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"Cystinosis Treatment Market Taxonomy"

On the basis of treatment, the cystinosis treatment market is segmented into:
Cysteine depleting therapy
Symptomatic therapy
Renal transplantation

On the basis of end users, the cystinosis treatment market is segmented into:
Hospitals
Specialty Clinics
Others

On the basis of region, the cystinosis treatment market is segmented into:
North America
Latin America
Europe
Asia Pacific
Middle East
Africa

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This release was published on openPR.