Cystinosis
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS trial_result · 2026-08-06Clinical Trial Results: Stem Cell Gene Therapy for Cystinosis [Phase 1, Phase 2]
A Phase 1/2 clinical trial evaluating a stem cell gene therapy for cystinosis completed, with results posted.
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CLINICAL_TRIALS research · 2026-04-20Clinical Trial Update: Use of Cysteamine in the Treatment of Cystinosis
Clinical trial update indicating recruitment for a study on cysteamine for cystinosis.
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CLINICAL_TRIALS trial_result · 2026-04-13Clinical Trial Update: PK and PD Study of NPI-001 and Cysteamine Bitartrate [Phase 1, Phase 2]
This clinical trial update describes a Phase 1/2 PK and PD study of NPI-001 in combination with cysteamine bitartrate for cystinosis, and th…
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CLINICAL_TRIALS trial_result · 2026-03-31Clinical Trial Update: Clinical and Biological Study of Sub-pigmentation During Infantile Cystinosis [NA]
The document summarizes the completion of a clinical and biological study on sub-pigmentation in infants with cystinosis.
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CLINICAL_TRIALS research · 2026-02-12Clinical Trial Update: European Cystinosis Cohort
The European Cystinosis cohort trial sponsored by INSERM in France is currently recruiting.
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CLINICAL_TRIALS trial_result · 2026-01-14Clinical Trial Update: Stem Cell Gene Therapy for Cystinosis [Phase 1, Phase 2]
The clinical trial update reports completion of the Phase 1/2 study evaluating stem cell gene therapy for cystinosis.
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CLINICAL_TRIALS research · 2026-01-06Clinical Trial Update: Cystinosis and Mitochondrial Metabolism [NA]
Clinical trial update related to cystinosis and kidney-related conditions. The study is not yet recruiting.
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CLINICAL_TRIALS trial_result · 2025-12-19Clinical Trial Update: A Cohort of Patients With Cystinosis : Compliance to Cysteamine and Neurological Complications [NA]
A completed cohort study in cystinosis reports on patient compliance with cysteamine therapy and associated neurological complications.
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CLINICAL_TRIALS trial_result · 2025-11-24Clinical Trial Update: Neuromuscular Characterisation in Late Adolescent and Adult Cystinosis Patients
The clinical trial evaluating neuromuscular characteristics in late adolescent and adult patients with cystinosis has completed.
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EMA approval · 2025-09-08EMA Authorised: Procysbi (mercaptamine bitartrate) — Chiesi Farmaceutici S.p.A
The European Medicines Agency authorised Procysbi (mercaptamine bitartrate) for the treatment of cystinosis, with Chiesi Farmaceutici S.p.A …
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EMA approval · 2025-08-08EMA Authorised: Cystadrops (mercaptamine hydrochloride) — Recordati Rare Diseases
EMA authorised Cystadrops, an eye drop containing mercaptamine hydrochloride, for the treatment of cystinosis; it carries orphan medicine st…
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EMA approval · 2024-12-11EMA Authorised: Cystagon (mercaptamine bitartrate) — Recordati Rare Diseases
The EMA authorised Cystagon (mercaptamine bitartrate) for the treatment of cystinosis, with Recordati Rare Diseases as the marketing authori…
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CLINICAL_TRIALS research · 2024-10-02Clinical Trial Update: The Effect of Exercise on Muscle Dysfunction in Cystinosis [NA]
Stanford University sponsored a clinical trial to assess whether exercise can improve muscle dysfunction in cystinosis. The trial has comple…
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CLINICAL_TRIALS research · 2024-08-22Clinical Trial Update: Development of Health-related Quality of Life Instrument for Patients With Cystinosis
This clinical trial update describes the development of a health-related quality of life instrument for patients with cystinosis. The work a…
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CLINICAL_TRIALS trial_result · 2024-07-03Clinical Trial Update: A Long-Term Follow-Up Study of Participants With Cystinosis Who Previously Received CTNS-RD-04
A long-term follow-up study is investigating outcomes in cystinosis patients who previously received CTNS-RD-04, with enrollment by invitati…
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CLINICAL_TRIALS trial_result · 2024-02-05Clinical Trial Update: Cystadrops in Pediatric Cystinosis Patients From Six Months to Less Than Two Years Old (SCOB2) [Phase 3]
Phase 3 clinical trial update for Cystadrops in pediatric cystinosis patients aged six months to less than two years completed.
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FDA label_change · 2022-02-25FDA Approved: CYSTARAN — Supplemental Application (Leadiant Biosciences, Inc.)
FDA approved a supplemental application for Cystaran (cysteamine hydrochloride) from Leadiant Biosciences, Inc. The action involves changes …
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FDA approval · 2020-08-19FDA Approved: CYSTADROPS — Original Application (RECORDATI RARE DISEASES, INC.)
FDA approves CYSTADROPS (cysteamine hydrochloride) from Recordati Rare Diseases for the treatment of corneal cystine crystal accumulation in…
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CLINICAL_TRIALS trial_result · 2018-07-24Clinical Trial Update: Long-Term Safety Follow-up Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) [Phase 3]
Phase 3 long-term safety follow-up of cysteamine bitartrate delayed-release capsules (RP103) in cystinosis has been completed, with results …
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CLINICAL_TRIALS trial_result · 2018-01-16Clinical Trial Update: Safety/Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis [Phase 3]
Phase 3 safety and effectiveness study of RP103 (cysteamine bitartrate delayed-release capsules) in cystinosis patients has completed and re…
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CLINICAL_TRIALS withdrawal · 2017-10-20Clinical Trial Update: Therapeutic Effect of Recombinant Human Growth Hormone (rhGH) on the Myopathy of Cystinosis [Phase 1, Phase 2]
A Phase 1/2 clinical trial evaluating recombinant human growth hormone for myopathy in cystinosis has been withdrawn.
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CLINICAL_TRIALS trial_result · 2017-10-06Clinical Trial Update: Kidney Transplantation in Patients With Cystinosis
The National Institute of Diabetes and Digestive and Kidney Diseases-sponsored trial evaluating kidney transplantation in cystinosis patient…
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FDA approval · 2017-09-24FDA 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 · 2017-07-02Clinical Trial Update: The Role of Susceptibility to Thrombosis in the Pseudotumor Cerebri of Nephropathic Cystinosis: A Case-Control Study
This completed case-control study examined whether susceptibility to thrombosis contributes to pseudotumor cerebri in patients with nephropa…
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CLINICAL_TRIALS trial_result · 2017-07-02Clinical Trial Update: Salivary Proteins in Disease and Health
An NIDCR-sponsored clinical trial update discusses salivary proteins in health and disease and lists studied conditions, including diabetes …