University of Health Sciences and Pharmacy in St. Louis has described estrogen-related receptor α (ERR) modulators reported to be useful for the treatment of diabetes, amyotrophic lateral sclerosis, heart failure, breast cancer, renal disorders, metabolic diseases, Alzheimer’s disease and Parkinson’s disease, among others.

Otsuka Pharmaceutical Co. Ltd. has described macrophage colony-stimulating factor 1 receptor (CSF1R; CD115; c-Fms) inhibitors reported to be useful for diagnosis and treatment of pain, infections, amyotrophic lateral sclerosis, multiple sclerosis, cardiovascular disorders, Alzheimer’s disease and Parkinson’s disease, among others.

Autism spectrum disorder, which affects around 1% of children worldwide, is as complex as the range of genetic and environmental factors that may contribute to it. Its complexity has made it difficult to identify effective treatments. Now researchers at Jazz Pharmaceuticals plc and Berand Neuropharmacology have demonstrated that a complex mixture extracted from Cannabis sativa shows therapeutic potential in one of the few preclinical models that mimics several characteristics of the disorder.

Aquinnah Pharmaceuticals Inc. has identified microtubule-associated protein tau (PHF-tau; MAPT) aggregation inhibitors reported to be useful for the treatment of Alzheimer's disease and frontotemporal dementia.

Vertex Pharmaceuticals Inc. has synthesized sodium channel protein type 10 subunit α (SCN10A; Nav1.8) blockers reported to be useful for the treatment of pain.

Autobahn Therapeutics Inc. has disclosed fatty acid amide (FAAH) cleavable prodrugs acting as sphingosine 1-phosphate S1P1 receptor (S1PR1; EDG1) and/or S1P5 receptor (S1PR5; EDG8) agonists reported to be useful for the treatment of multiple sclerosis, amyotrophic lateral sclerosis, Alzheimer’s disease, Parkinson’s disease, Huntington’s disease and epilepsy.

Researchers from Latus Bio Inc. have developed a novel adeno-associated virus (AAV) therapy for treating Huntington’s disease.