Niemann-pick Disease
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Sign up freeTop drugs studied for this disease
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EMA approval · 2026-07-09EMA Authorised: Zavesca (miglustat) — Advanz Pharma Limited
Authorization granted for Zavesca (miglustat) to Advanz Pharma Limited for Gaucher disease and Niemann-Pick diseases in the EU.
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CLINICAL_TRIALS research · 2026-04-22Clinical Trial Update: Retinal Hyperspectral Imaging in Neurodegenerative Diseases [NA]
A clinical trial is investigating retinal hyperspectral imaging for neurodegenerative diseases and dementia, currently recruiting participan…
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CLINICAL_TRIALS trial_result · 2026-02-19Clinical Trial Update: PREDIGA 2: Spanish Acronym of "Educational and Diagnostic Project for Gaucher and ASMD"
Clinical trial NCT05641103 (PREDIGA 2), an Educational and Diagnostic Project for Gaucher disease and acid sphingomyelinase deficiency, has …
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EMA approval · 2026-01-28EMA Authorised: Zavesca (miglustat) — Janssen Cilag International NV
EMA authorised Zavesca (miglustat) for Gaucher disease and Niemann-Pick diseases; Janssen Cilag International NV is the marketing authorisat…
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CLINICAL_TRIALS research · 2025-09-08Clinical Trial Update: UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells [Phase 1]
Phase 1 trial evaluating intrathecal umbilical cord blood-derived oligodendrocyte-like cells for several inherited metabolic brain diseases;…
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CLINICAL_TRIALS research · 2025-05-22Clinical Trial Update: National Multicentre Study of the Natural History of Acid Sphingo-myelinase Deficiency in Adults and Children
A national multicentre observational study investigating the natural history of acid sphingomyelinase deficiency (ASMD) in adults and childr…
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CLINICAL_TRIALS research · 2017-04-04Clinical Trial Update: Investigating Lysosomal Storage Diseases in Minority Groups
A clinical trial update from O & O Alpan LLC examining lysosomal storage disorders in minority populations, including Gaucher disease, Fabry…
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CLINICAL_TRIALS trial_result · 2015-07-30Clinical Trial Update: Tolerability and Safety Study of Recombinant Human Acid Sphingomyelinase in Acid Sphingomyelinase Deficiency Patients [Phase 1]
Phase 1 trial evaluating tolerability and safety of recombinant human acid sphingomyelinase in acid sphingomyelinase deficiency patients has…
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CLINICAL_TRIALS trial_result · 2015-03-19Clinical Trial Update: Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) [Phase 1]
Phase 1 safety study of recombinant human acid sphingomyelinase enzyme replacement therapy for adults with Niemann-Pick disease was terminat…
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CLINICAL_TRIALS research · 2008-03-04Clinical Trial Update: PET Scan of Brain Metabolism in Relation to Age and Disease
Completed clinical trial update describing PET imaging of brain metabolism across age and disease, including Alzheimer's disease, brain neop…