2024
Troriluzole rescues glutamatergic deficits, amyloid and tau pathology, and synaptic and memory impairments in 3xTg‐AD mice
Pfitzer J, Pinky P, Perman S, Redmon E, Cmelak L, Suppiramaniam V, Coric V, Qureshi I, Gramlich M, Reed M. Troriluzole rescues glutamatergic deficits, amyloid and tau pathology, and synaptic and memory impairments in 3xTg‐AD mice. Journal Of Neurochemistry 2024 PMID: 39214859, DOI: 10.1111/jnc.16215.Peer-Reviewed Original ResearchTau mouse modelsRestore cognitive deficitsAlzheimer's diseaseSynaptic vesiclesSV pool sizeMouse model of ADCognitive deficitsTau-related pathologyGlutamate releasePool sizeModel of ADHippocampal culturesGlutamate-modulating drugsLoss of glutamatergic synapsesBeta-amyloidGlutamate modulator riluzoleTau pathologyPresynaptic vesicular glutamate transporterVesicular glutamate transportersAD pathologyAccumulation of glutamateMouse modelTau modelStages of ADPresynaptic dysfunction
2023
P04 Taldefgrobep alfa: preclinical and clinical data supporting the phase 3 RESILIENT study in spinal muscular atrophy
Lair L, Qureshi I, Bechtold C, Heller L, Durham S, Campbell D, Marin J, Chen K, Coric V. P04 Taldefgrobep alfa: preclinical and clinical data supporting the phase 3 RESILIENT study in spinal muscular atrophy. Neuromuscular Disorders 2023, 33: s163. DOI: 10.1016/j.nmd.2023.07.381.Peer-Reviewed Original ResearchSpinal muscular atrophyMuscular atrophyClinical dataMuscle functionFlexor muscle functionMotor neuron lossRobust safety dataDownstream receptor signalingNon-ambulatory participantsAtrophic muscle fibersQuality of lifeSurvival motor neuron (SMN) proteinSMN upregulationMotor neuron geneNeuron lossMuscle weaknessSafety profileGastrocnemius weightPreclinical dataClinical studiesSafety dataMouse modelMuscle massNonclinical studiesMotor neuron protein