Duchenne muscular dystrophy is a severe and progressive disorder caused by mutations in the dystrophin (DMD) gene that lead to malfunction or absence of dystrophin. This protein stabilizes the sarcolemma and protects muscle cells during contraction.
Atai Therapeutics Inc. has identified 3-alkylamine indole compounds acting as 5-HT2A receptor agonists and reported to be potentially useful for the treatment of anxiety, eating and neurological disorders.
Miralinc Pharma Inc. has patented new heterocyclic selective histone deacetylase 6 (HDAC6) inhibitors reported to be useful for the treatment of type 2 Charcot-Marie-Tooth disease and chemotherapy-induced peripheral neuropathy, among others.
Humanwell Pharmaceutical US Inc. has synthesized new sigma non-opioid intracellular receptor 1 (SIGMAR1) antagonists reported to be useful for the treatment of cancer, pain, psychosis and substance abuse and dependence.
The central nervous system (CNS) in adult mammals has limited regeneration capacity, and traumatic injuries to the CNS usually lead to permanent functional impairment.
Centessa Pharmaceuticals plc recently detailed the discovery and preclinical characterization of a novel potent and selective orexin OX2 receptor agonist, ORX-142.
μ-Opioid receptor agonists have been described in a Miralogx LLC patent as potentially useful for anesthesia and sedation and the treatment of anxiety disorders, asthma, depression and pain.
Aquinnah Pharmaceuticals Inc. has identified new TAR DNA-binding protein 43 (TARDBP; TDP-43) aggregation and microtubule-associated protein tau (PHF-tau; MAPT) aggregation inhibitors reported to be useful for the treatment of amyotrophic lateral sclerosis (ALS) and Alzheimer’s disease.
Khalifa University and The University of Queensland presented preclinical data for a novel acid-sensing ion channel 1 (ASIC1) inhibitor, Hi1a, evaluated in models of multiple sclerosis (MS).
The adenosine A1 receptor (A1R) modulates ionic conductance in neuronal membrane and contributes to the anticonvulsant and neuroprotective role of the endogenous neuromodulator adenosine. Because A1Rs are present all throughout the body, the development of A1R modulation as antiseizure strategy may face difficulties such as side effects in the heart or other organs.