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Last Updated: 10/31/2025

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Found 422 publications

Correction: Miglustat ameliorates isoproterenol-induced cardiac fibrosis via targeting UGCG.

Correction: Miglustat ameliorates isoproterenol-induced cardiac fibrosis via targeting UGCG.

Journal: Molecular medicine (Cambridge, Mass.)
Published: September 23, 2025

Visual Recovery and Neurological Stabilization Following Miglustat Treatment in Pediatric CLN3 Disease.

Visual Recovery and Neurological Stabilization Following Miglustat Treatment in Pediatric CLN3 Disease.

Journal: Journal of child neurology
Published: September 18, 2025

Is Miglustat an Effective Treatment for CLN3 (Batten) Disease?

Is Miglustat an Effective Treatment for CLN3 (Batten) Disease?

Journal: Neurology
Published: September 09, 2025

Long-Term Open-Label Study Evaluating Oral Miglustat Treatment in Patients With Neuronal Ceroid Lipofuscinosis Type 3.

Long-Term Open-Label Study Evaluating Oral Miglustat Treatment in Patients With Neuronal Ceroid Lipofuscinosis Type 3.

Journal: Neurology
Published: September 09, 2025

The SGLT2 Inhibitor Dapagliflozin Disrupts the Cell Cycle at High Concentrations Without Altering Glycosphingolipid (De Novo)Biosynthesis.

The SGLT2 Inhibitor Dapagliflozin Disrupts the Cell Cycle at High Concentrations Without Altering Glycosphingolipid (De Novo)Biosynthesis.

Journal: International journal of molecular sciences
Published: August 20, 2025

Ketogenic Diet in Niemann-Pick Type C: Insights From a Case Report.

Ketogenic Diet in Niemann-Pick Type C: Insights From a Case Report.

Journal: International journal of developmental neuroscience : the official journal of the International Society for Developmental Neuroscience
Published: July 09, 2025

Cipaglucosidase alfa and miglustat for treatment of late-onset Pompe disease (LOPD): A therapeutics bulletin of the American College of Medical Genetics and Genomics (ACMG).

Cipaglucosidase alfa and miglustat for treatment of late-onset Pompe disease (LOPD): A therapeutics bulletin of the American College of Medical Genetics and Genomics (ACMG).

Journal: Genetics in medicine open
Published: July 07, 2025

Enzyme replacement therapies in adults with Pompe disease: from trials to real-world data.

Enzyme replacement therapies in adults with Pompe disease: from trials to real-world data.

Journal: Current opinion in neurology
Published: June 05, 2025

Biomarker Validation in NPC1: Foundations for Clinical Trials and Regulatory Alignment.

Biomarker Validation in NPC1: Foundations for Clinical Trials and Regulatory Alignment.

Journal: Journal of inherited metabolic disease
Published: June 01, 2025

Efficacy and safety of efavirenz in Niemann-Pick disease type C.

Efficacy and safety of efavirenz in Niemann-Pick disease type C.

Journal: Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics
Published: April 16, 2025

UDP-glucose ceramide glucosyltransferase promotes radioresistance via membrane reorganization to maintain redox balance in glioblastoma.

UDP-glucose ceramide glucosyltransferase promotes radioresistance via membrane reorganization to maintain redox balance in glioblastoma.

Journal: British journal of cancer
Published: April 03, 2025

Serum neurofilament light protein as a biomarker in Niemann-Pick disease, type C1.

Serum neurofilament light protein as a biomarker in Niemann-Pick disease, type C1.

Journal: Genetics in medicine open
Published: March 26, 2025
Showing 1-12 of 422

Last Updated: 10/31/2025