Trial of Cysteamine in Treatment of Corneal Cystine Accumulation in Cystinosis

局部应用半胱胺治疗胱氨酸病中角膜胱氨酸晶体堆积的试验 本研究将继续评价半胱胺滴眼液治疗胱氨酸病患者角膜中胱氨酸晶体的长期安全性和有效性。这些滴剂未上市销售，仅通过本研究提供。新患者可入组研究以获得这些药物。 胱氨酸病是一种遗传性疾病，会导致生长不良和肾脏疾病等。对肾脏和其他器官的损害被认为是由于胱氨酸在各种身体组织的细胞内的积累。这种化学物质也聚集在角膜中，角膜是覆盖在瞳孔和虹膜上的眼睛。10到20年后，一些患者的角膜变得如此拥挤，以至于表面可能变得不规则，偶尔会导致小的，痛苦的破裂。

Trial of Topical Cysteamine in the Treatment of Corneal Cystine Crystal Accumulation in Cystinosis This study will continue to evaluate the long term safety and effectiveness of cysteamine eye drops for treating cystine crystals in the corneas of patients with cystinosis. These drops are not sold commercially and are available only through this study. New patients may enroll in the study to obtain them. Cystinosis is an inherited disease that results in poor growth and kidney disease, among other things. The damage to the kidneys and other organs is thought to be due to accumulation of cystine inside the cells of various body tissues. This chemical also accumulates in the cornea-the covering of the eye over the pupil and iris. After 10 to 20 years, the corneas of some patients become so packed with crystals that the surfaces may become irregular, occasionally causing small, painful breaks.