Are patient-derived models of amyotrophic lateral sclerosis a game changer for novel drug discovery?

Abstract excerpt

INTRODUCTION: ALS drug discovery has long depended on model systems that incompletely capture human disease heterogeneity, aging, and TDP-43 proteinopathy. Patient-derived platforms have therefore emerged as increasingly important human-relevant complements to animal and molecular models. AREAS COVERED: This Critical Perspective examines when patient-derived ALS models genuinely change therapeutic decision-making...

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