Stowell, KathrynBurling, Sophie2025-06-022025-06-022025-01-29https://mro.massey.ac.nz/handle/10179/72980This research developed patient-derived multicellular disease models to advance diagnosis and drug discovery for neuromuscular and neurodegenerative diseases, such as malignant hyperthermia and motor neurone disease. A biobank of stem cells reprogrammed from patients facilitated the creation of “mini organoids” that replicate disease features. A muscle-on-chip platform utilised a microfluidic system for precise drug delivery, demonstrating potential to replace invasive diagnostics, and in ALS models, a novel repurposed drug effectively reduced key disease markers. The outcome is a scalable, patient-specific platform designed to enhance disease understanding, improve diagnostics, and expedite therapies for incurable and complex diseases.© The Authorstem cells, iPSC, muscle, amyotrophic lateral sclerosis, malignant hyperthermia, disease modelling, tissue engineering, drug discovery, diagnostics, neurodegenerative, neuromuscular disease, multicellular, microfluidics, organ-on-chipNeuromuscular diseasesPathogenesisInduced pluripotent stem cellsBiochipsNeuropharmacology3101 Biochemistry and cell biology320905 Neurology and neuromuscular diseases