Arimoclomol
Last Updated: 09/26/2026
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Found 68 publications
Current and emerging therapeutic advancements for Niemann-Pick disease type C: where are we now and what does the future hold?
Journal: Expert review of neurotherapeutics
Published: April 20, 2026
Long-term real-world safety and effectiveness of arimoclomol in individuals with NPC: Outcomes from the US early access program over a 4-year period.
Journal: Molecular genetics and metabolism
Published: April 13, 2026
Effects of Lysine Deacetylation Inhibition Alone or in Combination With Arimoclomol on TDP-43 Proteinopathy.
Journal: Journal of neurochemistry
Published: December 17, 2025
Arimoclomol in infants with Niemann-Pick disease type C: Results from the phase 2/3 open-label pediatric substudy.
Journal: Molecular genetics and metabolism reports
Published: November 20, 2025
Arimoclomol and levacetylleucine for the treatment of neurologic manifestations of Niemann-Pick disease type C: A therapeutics bulletin of the American College of Medical Genetics and Genomics (ACMG).
Journal: Genetics in medicine open
Published: September 29, 2025
Understanding Pharmacokinetic-Drug Interactions With Drugs Approved by the US Food and Drug Administration in 2024 to Better Manage the Risk of Drug Interactions With Concomitant Medications: A Review of Clinical Data From New Drug Applications.
Journal: Current therapeutic research, clinical and experimental
Published: August 07, 2025
Dose-dependent effects of Arimoclomol in ALS: insights from a network meta-analysis.
Journal: Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology
Published: July 18, 2025
Game Changers: Blockbuster Small-Molecule Drugs Approved by the FDA in 2024.
Journal: Pharmaceuticals (Basel, Switzerland)
Published: April 20, 2025
Long-term efficacy and safety of arimoclomol in Niemann-Pick disease type C: Final results of the phase 2/3 NPC-002 48-month open-label extension trial.
Journal: Molecular genetics and metabolism
Published: March 17, 2025
Efficacy results from a 12-month double-blind randomized trial of arimoclomol for treatment of Niemann-Pick disease type C (NPC): Presenting a rescored 4-domain NPC Clinical Severity Scale.
Journal: Molecular genetics and metabolism reports
Published: February 10, 2025
Therapeutic Potential of Arimoclomol Nanomicelles: In Vitro Impact on Alzheimer's and Parkinson's Pathology and Correlation with In Vivo Inflammatory Response.
Journal: ACS chemical neuroscience
Published: February 05, 2025
Quantitative muscle magnetic resonance imaging as a biomarker for inclusion body myositis in clinical trials: exploring the in vivo effects of arimoclomol.
Journal: Clinical and experimental rheumatology
Published: January 25, 2025
Last Updated: 09/26/2026