ORLANDO, Fla., March 22, 2022 /PRNewswire/ -- Leadiant Biosciences, Inc. has partnered with AllianceRx Walgreens Prime – one of the largest specialty and home delivery pharmacies in the U.S. – to be the exclusive specialty pharmacy and clinical support provider for its rare disease drug Cystaran.
Avrobio Inc's gene therapy for a rare lysosomal storage disorder showed potential durability in the first three patients more than one year after infusion.
LEBANON, N.J., Sept. 15, 2020 /PRNewswire/ -- Recordati Rare Diseases Inc., today announced that CYSTADROPS® (cysteamine ophthalmic solution) 0.37% is now available for prescription and distribution across the United States (U.S.). CYSTADROPS is a new, viscous eye drop solution that depletes cystine crystal deposits in the cornea of the eyes of people living with cystinosis. Cystinosis is a rare genetic condition present from birth that leads to the build-up of cystine crystals throughout the body, causing widespread tissue and organ damage and significant impact on the eyes.
Italy’s Recordati said on Tuesday it plans to seek Canadian approval for its drug Cystagon, which treats a rare kidney disease, in a potential blow to Horizon Pharma,, which makes the only drug approved for treatment in Canada.