Connecta Therapeutics SL has discovered 3-phenoxy-3-phenylpropanamine derivatives described as potentially useful for the treatment of fragile X and Rett syndrome.
Researchers from Cyclone Therapeutics Inc. and the Scripps Research Institute have disclosed tetracycline derivatives reported to be useful for the treatment of fragile X syndrome, rheumatoid arthritis and Alzheimer’s disease.
Medicxi Ventures’ asset-focused investment model has scored another hit, in a $450 million deal with Servier SA, for KER-0193, an oral small molecule that is ready for phase II development in the treatment of the inherited autism spectrum disorder, fragile X.
Fragile X syndrome (FXS), the most common inherited cause of intellectual disability and autism, is caused by silencing of the Fmr1 gene, leading to a lack of the FMRP protein, which regulates protein synthesis in neurons. One key pathway affected by FMRP loss is the metabotropic glutamate receptor 5 (mGluR5) signaling pathway, where activation of mGluR5 leads to excessive translation of several proteins involved in synaptic plasticity.
Quralis Corp. has entered into a number of agreements with the aim of advancing the treatment of fragile X syndrome, a genetic condition caused by a mutation of a single gene – fragile X messenger ribonucleoprotein 1 (FMR1) – on the X chromosome.
Growing evidence exists on regulation of the chloride importer solute carrier family 12 member 2 (SLC12A2), also known as NKCC1, as a therapeutic approach to treat neurological disorders. Altered expression of NKCC1 leads to impaired intracellular chloride levels in neurons and imbalance in the excitatory-inhibitory axis in the brain.
Fragile X syndrome (FXS) is the most common inherited form of intellectual disability and a leading monogenic cause of autism, yet effective treatments remain elusive. Previous work showed that N-methyl-D-aspartate receptors (NMDARs) play a prominent pathophysiological role in FXS and other neurodevelopmental disorders.
Neuren Pharmaceuticals Ltd. plans to progress NNZ-2591 to phase III trials following positive top-line phase II results in children with Phelan-McDermid syndrome, a genetic neurodevelopmental disorder for which there are no approved treatments.
Neuren Pharmaceuticals Ltd. plans to progress NNZ-2591 to phase III trials following positive top-line phase II results in children with Phelan-McDermid syndrome, a genetic neurodevelopmental disorder for which there are no approved treatments.