Sigma nonopioid intracellular receptor 1 (SIGMAR1) is a protein enriched in motor neurons (MNs), and mutations in its gene have been previously linked to various motor neuron diseases (MNDs). Researchers from Welab Barcelona and affiliated organizations recently presented preclinical data for two novel SIGMAR ligands, EST-79232 and EST-79376, being evaluated as potential candidates for the prevention of MN degeneration.

Nysnobio Inc. has received a grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to assist the advancement of the company’s lead parkin gene replacement therapy candidate, NB-001. The funding will support product manufacturing to enable IND safety studies in preparation for clinical trials evaluating NB-001 in Parkinson’s disease patients with biallelic loss-of-function mutations in the parkin gene, or parkin-PD.

Researchers from Nihon University and Aomori University have discovered a novel δ...

Spinocerebellar ataxia type 3 (SCA3) is the most common autosomal dominant hereditary ataxia worldwide, with no cure available. Intranuclear aggregation of the mutant expanded ATXN3 protein is a neuropathological hallmark of SCA3 linked to neurotoxicity.