Cystinosis
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS trial_result · 2017-05-24Clinical Trial Update: Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-Release Capsules (RP103) in Cystinosis [Phase 3]
Phase 3 open-label trial evaluating safety and superior effectiveness of cysteamine bitartrate delayed-release capsules (RP103) in Cystinosi…
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EMA approval · 2016-07-25EMA Refused: Dropcys (mercaptamine) — Lucane Pharma
The EMA refused marketing authorization for Dropcys (mercaptamine) by Lucane Pharma, for corneal diseases and cystinosis.
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FDA approval · 2015-09-09FDA Approved: PROCYSBI — Supplemental Application (Horizon Therapeutics USA, Inc.)
The FDA approved a supplemental application for PROCYSBI (cysteamine bitartrate) from Horizon Therapeutics USA, Inc.
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CLINICAL_TRIALS trial_result · 2015-08-26Clinical Trial Update: The Functional Consequences of the CTNS-deletion for the TRPV1-receptor in Cystinosis Patients [EARLY_Phase 1]
An early-phase clinical trial investigated how CTNS deletion affects TRPV1 receptor function in patients with cystinosis; the study has comp…
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CLINICAL_TRIALS trial_result · 2015-03-25Clinical Trial Update: Randomized Study of New Formulation Ophthalmic Cysteamine Hydrochloride for Corneal Cystine Accumulation in Patients With Cystinosis [NA]
It reports completion of a randomized trial evaluating a new formulation of ophthalmic cysteamine hydrochloride for corneal cystine accumula…
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CLINICAL_TRIALS trial_result · 2014-11-19Clinical Trial Update: Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis [Phase 3]
Phase 3 trial comparing cysteamine bitartrate delayed-release (RP103) to Cystagon in patients with cystinosis completed; results were posted…
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CLINICAL_TRIALS trial_result · 2014-10-28Clinical Trial Update: Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis [Phase 1, Phase 2]
A Phase 1/2 pilot trial compared RP103 with Cystagon for cystinosis to assess safety, tolerability, and pharmacokinetics/pharmacodynamics, w…
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CLINICAL_TRIALS trial_result · 2014-07-22Clinical Trial Update: Cysteamine Eye Drops to Treat Corneal Crystals in Cystinosis [Phase 2]
A Phase 2 clinical trial update reports results for cysteamine ophthalmic drops aimed at reducing corneal crystals in patients with cystinos…
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CLINICAL_TRIALS research · 2014-03-12Clinical Trial Update: Cure Cystinosis International Registry
This entry provides an update on the Cure Cystinosis International Registry (NCT01327807), a patient registry focused on cystinosis and rela…
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CLINICAL_TRIALS trial_result · 2013-10-09Clinical Trial Update: Study in Healthy Adults to Determine the Effect That Food Has on the Absorption and Delivery of the Drug Cystagon™ [NA]
A healthy volunteer study evaluated how food affects the absorption of Cystagon (cysteamine bitartrate); results were posted after completio…
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CLINICAL_TRIALS trial_result · 2012-06-20Clinical Trial Update: N Acetyl Cysteine for Cystinosis Patients [Phase 4]
A Phase 4 trial of N-acetylcysteine in cystinosis patients has completed.
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CLINICAL_TRIALS trial_result · 2008-03-04Clinical Trial Update: New Cysteamine Eye Drops Formulation to Treat Corneal Crystals in Cystinosis [Phase 1]
Phase 1 trial evaluating a new cysteamine eye drop formulation to treat corneal crystals in cystinosis completed.