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