Definitions

from Wiktionary, Creative Commons Attribution/Share-Alike License

  • n. A disorder caused by abnormal metabolism of cystine

Etymologies

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Examples

  • 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").

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  • Christy Greeley of Lincolnshire, Ill., said the drug Cystagon has saved lives of children with nephropathic cystinosis, including her 11-year-old son.

    Many 'Orphan Drugs' Get Expedited Review by FDA

  • Raptor's clinical programs include DR Cysteamine for the potential treatment of nephropathic cystinosis, non-alcoholic steatohepatitis "NASH", and Huntington's Disease.

  • For some background, on June 6, the company announced that on June 3 the last clinical trial patient visits were completed and final data analysis will commence this week on the Phase 3 clinical trial of its delayed-release oral formulation of DR Cysteamine in patients with cystinosis.

  • The pivotal Phase 3 clinical trial is designed as an outpatient study of the safety, tolerability, pharmacokinetics ("PK") and pharmacodynamics ("PD") of every 12-hour DR Cysteamine compared to every 6-hour immediate-release cysteamine bitartrate in cystinosis patients.

  • RPTP pivotal Phase 3 clinical trial of DR Cysteamine for the potential treatment of nephropathic cystinosis are expected by the end of July.

  • DR Cysteamine is designed for reduced dose frequency and improved tolerability, compared to immediate-release cysteamine, which is the current standard of care in cystinosis.

  • DR Cysteamine is Raptor's proprietary enteric-coated, microbead oral formulation of cysteamine bitartrate designed to potentially reduce dosing frequency and reduce gastrointestinal side effects associated with immediate-release cysteamine bitartrate, which is approved for sale by the FDA and European Medicines Agency to treat nephropathic cystinosis.

  • They interviewed a physician treating 25 persons for cystinosis which is a 5% of all US patients! and he was extremely positive on the results and on future approval

  • While cystinosis makes it tough to objectively assess adverse events since many of the side effects resolve on their own, the drawbacks of Cystagon are well documented nausea, vomiting, bad breath, etc., and thus I do not expect the FDA raising concerns with these points, given that patients seem very receptive to this twice daily formulation.

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