WHAT IS CYSTINOSIS
Cystinosis is a rare metabolic disease that affects approximately 500 people in the United States (mostly children) and about 2,000 people worldwide. This disease causes the amino acid “cystine” to accumulate in the cells of the body. Over time, the cystine damages various organs including the kidneys, liver, muscles, white blood cells, eyes, and central nervous system. Other complications include muscle wasting, growth loss, difficulty swallowing, and developmental delays.
Termed an “orphan disease,” Cystinosis is a disorder which has not been “adopted” by the pharmaceutical industry because it provides little financial incentive for the private sector to make and market new medications to treat it or prevent it.
2016 Q1 ISSUE COSTTREE CAPTURE. 15
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