Niemann-pick Disease Type
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CLINICAL_TRIALS research · 2026-04-23Clinical Trial Update: A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis [Phase 3]
This clinical trial update notes that oral Nizubaglustat (AZ-3102) is being studied in a Phase 3 trial for late-infantile and juvenile Niema…
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CLINICAL_TRIALS research · 2026-04-23Clinical Trial Update: A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC) [Phase 3]
Phase 3 study evaluating the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of Niemann-Pick diseas…
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CLINICAL_TRIALS research · 2026-04-20Clinical Trial Update: Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C
A clinical trial evaluating biochemical markers and clinical outcomes in Niemann-Pick Disease Type C is currently recruiting.
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CLINICAL_TRIALS research · 2026-04-20Clinical Trial Update: Establishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type C
A clinical trial aiming to establish a genomic and phenotypic database for Niemann-Pick disease type C, currently recruiting.
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CLINICAL_TRIALS research · 2026-04-03Clinical Trial Update: A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C [Phase 3]
A pivotal Phase 3 trial evaluating N-Acetyl-L-Leucine for Niemann-Pick disease type C is currently recruiting.
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CLINICAL_TRIALS research · 2026-03-19Clinical Trial Update: A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease [Phase 2]
A Phase 2 clinical trial is evaluating the safety and efficacy of Nizubaglustat (AZ-3102) in patients with GM2 gangliosidosis and Niemann-Pi…
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EMA approval · 2026-03-03EMA Authorised: Aqneursa (L-acetylleucine) — Intrabio Ireland Limited
The European Medicines Agency authorised Aqneursa (L-acetylleucine) by Intrabio Ireland Limited for Niemann-Pick disease, type C; it is desi…
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CLINICAL_TRIALS trial_result · 2026-01-07Clinical Trial Update: MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis [Phase 2]
Phase 2 trial update describing allogeneic hematopoietic cell transplantation for metabolic disorders and severe osteopetrosis, with the stu…
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CLINICAL_TRIALS trial_result · 2025-12-12Clinical Trial Update: Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C [Phase 1, Phase 2]
Phase 1/2 trial evaluating intravenous VTS-270 for infantile liver disease in Niemann-Pick Disease, Type C has completed and results have be…
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CLINICAL_TRIALS research · 2025-10-31Clinical Trial Update: A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency (ASMD)
This is an ongoing real-world, long-term safety and immunogenicity study of olipudase alfa in infants under 2 years with acid sphingomyelina…
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CLINICAL_TRIALS research · 2025-09-12Clinical Trial Update: ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
This clinical trial update describes ScreenPlus, a comprehensive, multi-disorder newborn screening program that is currently enrolling by in…
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EMA approval · 2025-07-25New treatment for Niemann-Pick type C disease
The EMA announced a new treatment, Aqneursa, for Niemann-Pick disease type C, which significantly improves neurological signs, symptoms and …
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CLINICAL_TRIALS research · 2025-05-18Clinical Trial Update: Study of the Prevalence of Acid Sphingomyelinase Deficiency/Niemann Pick AB and B Disease in Patients With Diffuse Interstitial Lung Disease [NA]
A clinical trial to determine the prevalence of acid sphingomyelinase deficiency (Niemann-Pick disease types A and B) in patients with diffu…
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CLINICAL_TRIALS trial_result · 2025-03-30Clinical Trial Update: Safety and Efficacy of Miglustat in Chinese NPC Patients [Phase 4]
Phase 4 study in Chinese Niemann-Pick Disease Type C patients evaluated miglustat for safety and efficacy. The trial has completed.
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CLINICAL_TRIALS research · 2025-03-20Clinical Trial Update: Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients with GM2 Gangliosidosis or Niemann-Pick Type C Disease [Phase 2]
Phase 2 study evaluating safety, tolerability, pharmacokinetics and pharmacodynamics of oral AZ-3102 in GM2 gangliosidosis and Niemann-Pick …
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FDA approval · 2025-03-14FDA Approved: MIGLUSTAT — Original Application (Navinta LLC)
The FDA approved the Original Application (ANDA) for miglustat by Navinta LLC, granting a generic approval for the product.
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CLINICAL_TRIALS trial_result · 2025-02-04Clinical Trial Update: Miglustat in Niemann-Pick Type C Disease [Phase 2]
Phase 2 trial of miglustat for Niemann-Pick disease type C has completed.
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FDA approval · 2024-12-20FDA Approved: XENPOZYME — Supplemental Application (Genzyme Corporation)
FDA approved a supplemental application for XENPOZYME (olipudase alfa-RPCP) from Genzyme Corporation.
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CLINICAL_TRIALS research · 2024-08-16Clinical Trial Update: Early Access Program With Arimoclomol in US Patients With NPC
The document reports an Early Access Program update for arimoclomol in US patients with Niemann-Pick Disease Type C. It describes ongoing ac…
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CLINICAL_TRIALS trial_result · 2024-01-17Clinical Trial Update: Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System [Phase 3]
Phase 3 trial of Adrabetadex for Niemann-Pick Type C disease has been terminated; results posted.
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CLINICAL_TRIALS trial_result · 2023-12-26Clinical Trial Update: Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1 [Phase 2, Phase 3]
Open-label study of VTS-270 in neurologic manifestations of Niemann-Pick disease type C1 was terminated; results have been posted.
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FDA approval · 2023-12-05FDA Approved: XENPOZYME — Supplemental Application (Genzyme Corporation)
FDA approved a supplemental application for XENPOZYME (olipudase alfa-rpcp) from Genzyme Corporation. This supplemental approval concerns th…
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CLINICAL_TRIALS trial_result · 2023-11-28Clinical Trial Update: N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC) [Phase 2]
Phase 2 trial update for N-Acetyl-L-Leucine in Niemann-Pick Disease, Type C (NPC) completed with results posted.
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CLINICAL_TRIALS trial_result · 2023-08-29Clinical Trial Update: Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease [Phase 2, Phase 3]
Clinical trial update for adrabetadex in Niemann-Pick disease type C1 (NPC1) has terminated after Phase 2/3; results posted were in 2023.
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CLINICAL_TRIALS trial_result · 2023-06-09Clinical Trial Update: Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C [Phase 2, Phase 3]
A prospective Phase 2/3 trial of arimoclomol in Niemann-Pick Disease Type C has completed and results have been posted.