Tevard Biosciences Inc. has presented new preclinical data on the use of therapeutic suppressor tRNAs (suptRNAs) for the treatment of Duchenne muscular dystrophy (DMD) and dilated cardiomyopathy (DCM). The data show potent restoration of full-length functional proteins in models of DMD and DCM caused by titin truncations (DCM-TTNtv).
Hager Biosciences LLC has identified orexin receptor and/or κ-opioid receptor dual antagonists reported to be useful for the treatment of cancer, psychiatric, neurological and cardiovascular disorders.
Researchers from Novartis AG reported preclinical efficacy data on VHB-937, an agonist human monoclonal antibody targeting TREM2 in models of neuroinflammation.
Is there a link between cellular senescence and multiple sclerosis (MS) progression? Several presentations at this year’s European Committee for Treatment and Research in Multiple Sclerosis 2025 (ECTRIMS 2025) conference, which ends today in Barcelona, addressed this question.
Neushen Therapeutics Inc. has patented NLRP3 inflammasome inhibitors reported to be useful for the treatment of Alzheimer’s disease, Parkinson’s disease, multiple sclerosis, amyotrophic lateral sclerosis and Huntington’s disease.
The FDA has awarded orphan drug designation to Cure Rare Disease’s CRD-003 for the treatment of limb-girdle muscular dystrophy type R9 (LGMD2i/R9), a congenital muscular dystrophy caused by biallelic mutations in the FKRP gene.
The Royal College of Psychiatrists in the U.K. has published its first-ever guidance to support research into psychedelic drugs as therapies for conditions including treatment-resistant depression, substance abuse disorders and posttraumatic stress disorder, saying that in a fast-moving field there is a risk of jumping ahead of the evidence.
Humanwell Healthcare (Group) Co. Ltd. has divulged P2X purinoceptor 7 (P2RX7; P2X7) antagonists reported to be useful for the treatment of pain, inflammatory, neurological and immunological disorders.
Miralogx LLC and Mira Pharmaceuticals Inc. have jointly disclosed new cannabinoid analogues acting as monoamine oxidase-A (MAO-A) and/or MAO-B inhibitors reported to be useful for the treatment of epilepsy, anxiety disorders and pain.