References in periodicals archive ?
Corneal crystals in nephropathic cystinosis: natural history and treatment with cysteamine eyedrops.
patient with nephropathic cystinosis will be denied access to PROCYSBI based on inability to pay.
In December 2007, Raptor obtained an exclusive, worldwide license from the University of California, San Diego for the development of RP103 for nephropathic cystinosis and for cysteamine for other potential indications including Huntington's Disease currently in a Phase 2/3 clinical trial in France and non-alcoholic steatohepatitis ("NASH") currently in a Phase 2b clinical trial in the U.
Raptor" or the "Company") (Nasdaq: RPTP), today announced the publication of results from a Phase 2a clinical trial of a prototype formulation of its proprietary delayed-release cysteamine bitartrate ("DR Cysteamine") in patients with nephropathic cystinosis ("cystinosis").
Thus, even advanced lesions of diabetic nephropathy may be reversed, but only over a period of time (1 decade) approaching the duration of diabetes necessary to develop nephropathic lesions (1-2 decades) (20).
Raptor Pharmaceutical) announced that the Office of Orphan Product Development at the Food and Drug Administration (FDA) has granted the Company orphan drug exclusivity for PROCYSBI (cysteamine bitartrate) delayed-release capsules for the management of nephropathic cystinosis patients of the age six and above.
The therapy of nephropathic cystinosis involves treatment with the cystine-depleting agent cysteamine (2).
This team has invested in 34 pharmaceutical and medical device products through two HC Royalty investment funds including LYRICA,Oracea, Cetrotide, and PROCYSBI (TM), an orphan drug recently approved by the FDA for the treatment of nephropathic cystinosis.
Cysteamine therapy for children with nephropathic cystinosis.
About Nephropathic Cystinosis Nephropathic cystinosis comprises 95% of cystinosis, a rare, life-threatening metabolic lysosomal storage disorder that causes toxic accumulation of cystine in all cells, tissues, and organs in the body.
Raptor" or the "Company") (Nasdaq: RPTP), today announced results from a Phase 2b clinical trial of its proprietary delayed-release cysteamine bitartrate ("DR Cysteamine") in patients with nephropathic cystinosis ("cystinosis").
A novel gene encoding an integral membrane protein is mutated in nephropathic cystinosis.