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
2020
Assessing Effects of BHV-0223 40 mg Zydis Sublingual Formulation and Riluzole 50 mg Oral Tablet on Liver Function Test Parameters Utilizing DILIsym
Longo D, Shoda L, Howell B, Coric V, Berman R, Qureshi I. Assessing Effects of BHV-0223 40 mg Zydis Sublingual Formulation and Riluzole 50 mg Oral Tablet on Liver Function Test Parameters Utilizing DILIsym. Toxicological Sciences 2020, 175: 292-300. PMID: 32040174, PMCID: PMC7253195, DOI: 10.1093/toxsci/kfaa019.Peer-Reviewed Original ResearchConceptsOral tabletsALT elevationLiver toxicityClinical dataFirst-pass liver metabolismLiver function test parametersFirst-pass hepatic metabolismLiver toxicity riskAlanine transaminase levelsRelative risk reductionLower overall doseAmyotrophic lateral sclerosisALT levelsTransaminase levelsLiver exposureSublingual formulationHepatic metabolismLateral sclerosisRiluzoleLiver metabolismULNInterindividual variabilityOverall doseHepatotoxicity potentialOxidative stress