A recent study in PLoS One by researchers from Cincinnati Children’s Hospital Medical Center has evaluated the preclinical long-term safety of human A gamma-globin gene-carrying GbGM LV in wild-type mice.

Researchers from Royal Children's Hospital, the University of Melbourne and affiliated organizations published data from a study that aimed to investigate the potential of benfotiamine, which is a lipid soluble precursor to thiamine, for the treatment of inflammation related dystrophic pathology in patients with Duchenne muscular dystrophy (DMD).

After receiving Orphan Drug Designation earlier this year, Satellos Bioscience Inc. announced that the FDA has granted Rare Pediatric Disease Designation to SAT-3247 for the potential treatment of Duchenne muscular dystrophy (DMD).

Researchers from Indiana University have presented data from a study that aimed to assess the potential of suppressing GABA metabolism through inhibition of its degradation enzyme, GABA aminotransferase (GABA-AT), as novel analgesic strategy. The effect of next-generation GABA-AT inhibitor OV-329 was assessed in C57BL/6J mice that were intraperitoneally injected with paclitaxel to generate neuropathic nociception. Treatment with OV-329 attenuated the development and maintenance of paclitaxel-induced mechanical hypersensitivity.

Researchers at Leiden University have synthesized semisynthetic guanidino lipoglycopeptides with in vitro and in vivo activity gram-positive bacteria, including methicillin-resistant and vancomycin-resistant Staphylococcus aureus.

Infection or cure? Scientists from Tel Aviv University and the University of Glasgow genetically modified the Toxoplasma gondii to bring a protein inside neurons. The novelty of using a protozoan that can travel from the gut to parasitize the CNS contrasts with the possibility of causing a disease. The scientists are already working on how to avoid it.