2025
Pridopidine in early-stage manifest Huntington’s disease: a phase 3 trial
Reilmann R, Feigin A, Rosser A, Kostyk S, Saft C, Cohen Y, Schuring H, Hand R, Tan A, Chen K, Feng W, Navon-Perry L, Cruz-Herranz A, Syltevik C, Boot D, Squitieri F, Kayson E, Mehra M, Goldberg Y, Geva M, Hayden M. Pridopidine in early-stage manifest Huntington’s disease: a phase 3 trial. Nature Medicine 2025, 1-10. PMID: 40913168, DOI: 10.1038/s41591-025-03920-3.Peer-Reviewed Original ResearchLeast-squares mean differenceTotal functional capacityComposite Unified Huntington’s Disease Rating Scale,Phase 3 trialHuntington's diseasePlacebo-controlled phase 3 trialSigma-1 receptor agonistUnified Huntington's Disease Rating ScaleHuntington's Disease Rating ScaleTolerability profileDouble-blindManifest Huntington's diseaseReceptor agonistsSecondary endpointsSubgroup of participantsAntidopaminergic medicationsPridopidineSymptomatic treatmentSigma-1Rating ScaleDisease Rating ScaleOverall populationUnmet needsFunctional capacityNeurodegenerative disordersThe Safety Profile of Pridopidine, a Novel Sigma-1 Receptor Agonist for the Treatment of Huntington’s Disease
Goldberg Y, Navon-Perry L, Cruz-Herranz A, Chen K, Hecker-Barth G, Spiegel K, Cohen Y, Niethammer M, Tan A, Schuring H, Geva M, Hayden M. The Safety Profile of Pridopidine, a Novel Sigma-1 Receptor Agonist for the Treatment of Huntington’s Disease. CNS Drugs 2025, 39: 485-498. PMID: 40055280, PMCID: PMC11982116, DOI: 10.1007/s40263-025-01171-x.Peer-Reviewed Original ResearchSafety profileAdverse eventsAntidopaminergic medicationsOpen-label extension studySigma-1Sigma-1 receptor agonistCytosine-adenine-guaninePlacebo-controlled studySpectrum of disease severityAnalysis of pooled dataTotal functional capacityFirst-in-classRecommended human doseSigma-1 receptorTreatment of Huntington's diseaseUnmet medical needLong-term useDouble-blindReceptor agonistsPridopidineHuman doseSafety signalsEffective treatmentPlaceboExtension study
2019
Novel radioligands for imaging sigma-1 receptor in brain using positron emission tomography (PET)
Lan Y, Bai P, Chen Z, Neelamegam R, Placzek M, Wang H, Fiedler S, Yang J, Yuan G, Qu X, Schmidt H, Song J, Normandin M, Ran C, Wang C. Novel radioligands for imaging sigma-1 receptor in brain using positron emission tomography (PET). Acta Pharmaceutica Sinica B 2019, 9: 1204-1215. PMID: 31867166, PMCID: PMC6900558, DOI: 10.1016/j.apsb.2019.07.002.Peer-Reviewed Original ResearchPositron emission tomographySigma-1 receptorCentral nervous systemPET/CT imagingEmission tomographyOrgans of micePositron emission tomography tracersNeuropsychiatric disordersNon-invasive quantificationBrain regionsNoninvasive toolSigma-1RadioligandDisease pathophysiologyNervous systemNovel radioligandMiceMouse brainPathological conditionsBrainReceptorsTomographyIntracellular proteinsTarget validationPET/CT
2017
Structural Perspectives on Sigma-1 Receptor Function
Alon A, Schmidt H, Zheng S, Kruse A. Structural Perspectives on Sigma-1 Receptor Function. Advances In Experimental Medicine And Biology 2017, 964: 5-13. PMID: 28315261, DOI: 10.1007/978-3-319-50174-1_2.Peer-Reviewed Original ResearchConceptsER-resident transmembrane proteinCarboxy-terminal domainDrug-like small moleculesOverall foldsLigand moleculesTransmembrane proteinsMolecular structureCrystal structureHuman diseasesLigand recognitionMolecular viewReceptor arrangementSmall moleculesProtomersSigma-1LigandReceptor structureProteinMoleculesReceptor functionSigma-1 receptorStructureOligomerizationReceptorsCrystal
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