Niemann-pick Disease Type
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS trial_result · 2023-02-22Clinical Trial Update: VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease [Phase 2, Phase 3]
Clinical trial update for VTS-270 in Niemann-Pick Disease Type C1 (NPC1) across Phase 2 and Phase 3, with completed status and results poste…
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EMA withdrawal · 2023-02-08EMA Application withdrawn: Miplyffa (arimoclomol citrate) — Orphazyme A/S
The EMA has withdrawn the application for Miplyffa (arimoclomol citrate) by Orphazyme A/S for Niemann-Pick Disease Type C.
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CLINICAL_TRIALS trial_result · 2022-05-24Clinical Trial Update: Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency [Phase 2, Phase 3]
Phase 2/3 trials evaluated the efficacy, safety, pharmacodynamics, and pharmacokinetics of olipudase alfa in patients with acid sphingomyeli…
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EMA approval · 2022-05-20First therapy to treat two types of Niemann-Pick disease, a rare genetic metabolic disorder
EMA announces the first therapy approved to treat two types of Niemann-Pick disease, a rare genetic metabolic disorder.
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CLINICAL_TRIALS trial_result · 2021-08-10Clinical Trial Update: Safety and Efficacy of Intravenous Trappsol Cyclo (HPBCD) in Niemann-Pick Type C Patients [Phase 1, Phase 2]
A completed Phase 1/2 clinical trial evaluating intravenous Trappsol Cyclo (HPBCD) for Niemann-Pick disease type C1, focusing on safety and …
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FDA approval · 2020-08-06FDA Approved: YARGESA — Original Application (Edenbridge Pharmaceuticals LLC.)
FDA approved Edenbridge Pharmaceuticals' original ANDA for YARGESA, a generic version of miglustat. Miglustat is the active ingredient and i…
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FDA approval · 2019-11-14FDA Approved: MIGLUSTAT — Supplemental Application (ANI Pharmaceuticals, Inc.)
The FDA approved ANI Pharmaceuticals' supplemental application for miglustat (MIGLUSTAT) under ANDA 208342.
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FDA approval · 2019-06-26FDA Approved: ZAVESCA — Supplemental Application (Actelion Pharmaceuticals US, Inc.)
FDA approved a supplemental application for ZAVESCA (miglustat) from Actelion Pharmaceuticals US, Inc., potentially updating the product lab…
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FDA approval · 2018-04-17FDA Approved: MIGLUSTAT — Original Application (ANI Pharmaceuticals, Inc.)
The FDA approved ANI Pharmaceuticals' miglustat original-application product (generic miglustat). It is indicated for Niemann-Pick disease t…
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CLINICAL_TRIALS trial_result · 2017-07-02Clinical Trial Update: Saccadic Eye Movements in Patients With Niemann-Pick Type C Disease [Phase 1]
Phase 1 clinical trial completed evaluating saccadic eye movements in patients with Niemann-Pick disease type C.
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CLINICAL_TRIALS trial_result · 2017-05-18Clinical Trial Update: A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C
Prospective non-therapeutic observational study in patients with Niemann-Pick Disease Type C; the trial status is completed.
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CLINICAL_TRIALS trial_result · 2017-01-13Clinical Trial Update: Screening of Niemann-Pick Disease, Type C in a Psychiatric Population [NA]
A clinical trial screening Niemann-Pick disease type C in a psychiatric adult population (NCT02841358) sponsored by University Hospital, Gre…
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FDA approval · 2016-01-15FDA Approved: ZAVESCA — Supplemental Application (Actelion Pharmaceuticals US, Inc.)
The FDA approved a supplemental application for ZAVESCA (miglustat) from Actelion Pharmaceuticals US, updating the labeling.
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CLINICAL_TRIALS trial_result · 2016-01-06Clinical Trial Update: Longitudinal Study of Cognition With Niemann-Pick Disease, Type C
This entry notes the completion of a longitudinal cognitive study in Niemann-Pick disease, type C. No treatment results are provided in the …
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CLINICAL_TRIALS trial_result · 2013-01-04Clinical Trial Update: Application of Miglustat in Patients With Niemann-Pick Type C [Phase 3]
Phase 3 trial update of miglustat in patients with Niemann-Pick disease type C; the study is reported as completed.
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CLINICAL_TRIALS trial_result · 2012-07-12Clinical Trial Update: Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine [Phase 1, Phase 2]
A Phase 1/2 clinical trial evaluating the safety and potential efficacy of N-acetylcysteine in Niemann-Pick disease Type C has completed, wi…