{"id":49771,"date":"2022-10-18T20:01:37","date_gmt":"2022-10-18T18:01:37","guid":{"rendered":"https:\/\/pharma-trend.com\/en\/unique-human-neuromuscular-junction-assay-establishes-skeletal-muscle-as-a-key-player-in-als\/"},"modified":"2022-10-18T20:01:37","modified_gmt":"2022-10-18T18:01:37","slug":"unique-human-neuromuscular-junction-assay-establishes-skeletal-muscle-as-a-key-player-in-als","status":"publish","type":"post","link":"https:\/\/pharma-trend.com\/en\/unique-human-neuromuscular-junction-assay-establishes-skeletal-muscle-as-a-key-player-in-als\/","title":{"rendered":"Unique human neuromuscular junction assay establishes skeletal muscle as a key player in ALS"},"content":{"rendered":"
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ORLANDO, Fla.–(BUSINESS WIRE)–#ALS<\/a>–In a recent study published in Biomaterials<\/a>, a team of researchers led by Hesperos Chief Scientist Dr. J. Hickman established a significant finding to better understand ALS etiology and potential new treatment targets. Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig\u2019s disease, is a debilitating disease that expresses as progressive muscle weakness and loss of motor function typically leading to death within 2-5 years of diagnosis. Researchers continue to work to understand what is driving that dysfunction but progress has been slow primarily due to the limited availability of human-based models. This study used human induced pluripotent stem cells in a microphysiological system (MPS) that compared wild type (healthy) cells against patient derived mutant (diseased) cells. Specifically, the team looked at the relative contributions of diseased skeletal muscle and motoneurons on disease pathophysiology.\n<\/p>\n

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\nResults from the MPS disease model indicated a significant reduction in the number of functional neuromuscular junctions (NMJ\u2019s), stability of those NMJ\u2019s that did form, and an increased fatigue index which are all clinically relevant readouts. While the root cause of ALS still remains unresolved, the study produced findings that suggest diseased skeletal muscle plays a significant role in NMJ signal disruption, independent of motoneurons. Ultimately, these results suggest that therapeutically targeting the diseased muscle may be more critical than targeting diseased motoneurons to slow disease progression in addition to further validating the human-on-a-chip platform for further research.\n<\/p>\n