Mucopolysaccharidosis type III (MPS III), also known as Sanfilippo syndrome, is a congenital metabolic disorder that leads to the accumulation of partially degraded heparan sulfate, which triggers neurodegeneration.
Shanghai Huilun Pharmaceutical Co. Ltd. has identified sodium channel protein type 10 subunit α (SCN10A; Nav1.8) blockers reported to be useful for the treatment of pain, multiple sclerosis, Charcot-Marie-Tooth disease, urinary incontinence, cough and arrhythmia.
Atamyo Therapeutics SAS has received clinical trial application (CTA) authorizations in Italy and France for ATA-200, its gene therapy for the treatment of γ-sarcoglycan related limb-girdle muscular dystrophy type 2C/R5 (LGMD2C/R5).
Pannex Therapeutics Inc. has divulged pannexin-1 (PANX1; MRS1) inhibitors reported to be useful for the treatment of neuropathic pain and opioid dependency.
Brise Pharmaceutical (Shanghai) Co. Ltd. has identified calcitonin gene-related peptide (CGRP) receptor antagonists reported to be useful for treatment of cerebrovascular and vascular disorders.
Nurexone Biologic Inc. has signed a strategic service agreement with Vivox Ltd. for large-scale animal testing of Exopten therapy for spinal cord injury (SCI).
To address the need for novel therapeutic candidates against Duchenne muscular dystrophy (DMD), investigators at Mitorx Therapeutics Ltd. developed a library of novel small-molecule mitochondriotropic agents.
Galectin-3 (Gal-3) is used as a marker of activated microglia, and thus a protein that drives inflammation. Patients with Alzheimer’s disease (AD) have shown increased levels of Gal-3 in their cerebrospinal fluid (CSF) compared to healthy controls, as well as increased expression in their brain tissue.
Vaccines that target α-synuclein have a lot of potential for the treatment of Parkinson’s disease (PD) and other synucleinopathies. Tridem Bioscience GmbH & Co. KG has developed the proprietary WISIT vaccine technology.