amyotrophic lateral sclerosis

[Scientific Reports] Scientists demonstrated that protein disulphide isomerase (PDI) was protective against mutant Fused in Sarcoma (FUS). In neuronal cell line and primary cultures, PDI restored defects in nuclear import, prevented the formation of mutant FUS inclusions, inhibited Golgi fragmentation, ER stress, ER-Golgi transport defects, and apoptosis.

[Communications Biology] Using both human induced pluripotent stem cells and mouse models, researchers found that FUS-ALS causative mutations affect the activity of two relevant RNA-binding protein with important roles in neuronal RNA metabolism: HuD/ELAVL4 and FMRP.

[UC San Diego Health] The governing Board of the California Institute for Regenerative Medicine has approved a trio of awards, totaling approximately $4 million, to University of California San Diego School of Medicine researchers to advance studies of new stem cell-based treatments for multiple types of cancer and amyotrophic lateral sclerosis.

[AB Science SA] AB Science SA announced that it has received the first authorization to resume patient enrollment in the confirmatory Phase III study of masitinib in amyotrophic lateral sclerosis (ALS). Masitinib is a orally administered tyrosine kinase inhibitor that targets mast cells and macrophages, important cells for immunity, through inhibiting a limited number of kinases.

[PharmaTher Holdings Ltd.] PharmaTher Holdings Ltd. announced that the FDA has granted orphan drug designation for ketamine in the treatment of Amyotrophic Lateral Sclerosis, also known as Lou Gehrig's disease.

[Nature Communications] Investigators generated sensorimotor organoids containing physiologically functional neuromuscular junctions and applied the model to different subgroups of amyotrophic lateral sclerosis.