Alterations in the GABAergic pathway in the brain contribute to the pathophysiology of neurodevelopmental disorders, including autism spectrum disorder and Angelman syndrome. Targeting the GABA-A receptor α5 subunit (GABRA5) with positive allosteric modulators represents a new therapeutic strategy for the treatment of these conditions.

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.

Neuronos Ltd., a subsidiary of Beyond Air Inc., has announced BA-102 has been awarded U.S. orphan drug designation for the treatment of Phelan-McDermid syndrome.

Writing in Molecular Therapy Nucleic Acids, researchers hypothesized that using poly(A) tail mimetics to enhance mRNA expression from haploinsufficiency-associated genes could be a disease-modifying treatment strategy.