Cystinosis is a multisystemic lysosomal storage disorder caused by pathogenic variants in CTNS, the gene encoding cystinosin, a lysosomal transmembrane cystine transporter. In patients with cystinosis ...

Cystagon (cysteamine bitartrate) is a prescription drug used to treat nephropathic cystinosis in children and adults. This is a rare genetic condition that causes a buildup of cystine (an amino acid) ...

Cystinosis is an autosomal recessive lysosomal storage disorder caused by biallelic mutations in the CTNS gene, which encodes the seven-transmembrane lysosomal exporter cystinosin. Loss of cystinosin ...

Leadiant Biosciences, Inc. today announced that Health Canada has approved Cysklar® (cysteamine ophthalmic solution) 0.44%, a topical eye drop solution indicated for the treatment of corneal cystine ...

“Cystinosis is a rare genetic condition that very few people have heard of. It causes a natural substance called cystine to build up in the body’s cells over time. Normally, cells naturally remove ...

Procysbi (cysteamine) is a prescription drug approved to treat nephropathic cystinosis in adults and some children. It does not cure the underlying genetic disorder, but it can ease symptoms and ...