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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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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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 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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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…