An Open-Label, Phase III Study to Assess the Efficacy and Safety of Cysteamine Ophthalmic Solution 0.55% in Japanese ...
Introduction Cystinosis is a rare autosomal recessive lysosomal storage disorder caused by mutations in the cystinosin gene (CTNS) on chromosome 17.1,2 CTNS encodes a membrane transporter protein, cystinosin, which exports lysosomal cystine into the …