Cystinosis Treatment: Advancements and Challenges in Managing a Rare Genetic Disorder
The primary goal of Cystinosis Treatment is to reduce the accumulation of cystine crystals in the body and prevent or delay organ damage. The mainstay of treatment is the drug cysteamine, which helps to break down cystine crystals and reduce their buildup within cells. Cysteamine is available in oral and eye drop formulations, and patients typically require lifelong treatment.

Cystinosis is a rare genetic disorder characterized by the accumulation of the amino acid cystine within cells, leading to the formation of cystine crystals. These crystals can damage various organs and tissues throughout the body, particularly the kidneys, eyes, and muscles. The most common form of cystinosis, nephropathic cystinosis, typically manifests in infancy or early childhood and can lead to kidney failure if left untreated.

Cystinosis Treatment Market

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