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).

Medannex Ltd. has been awarded an Innovate UK grant to fund a £313,000 (US$403,000) project developing a new treatment for pediatric osteosarcoma. Innovate UK will fund £231,000 (US$297,000) of the project costs, with the remainder financed by Medannex.

One of the most common causes of adverse neurological disabilities in newborns is neonatal hypoxic-ischemic encephalopathy. Therapeutic hypothermia (TH) is the standard therapy, but it is not efficient in all cases. Cannabinoids have raised interest as therapeutics as they are neuroprotective.

“Prenatal therapies are the next disruptive technologies in health care, which will advance and shape the future of patient care in the 21st century,” said Graça Almeida-Porada, a professor at the Fetal Research and Therapy Center of the Wake Forest Institute for Regenerative Medicine in Winston-Salem, North Carolina. At the American Society of Gene & Cell Therapy (ASGCT) annual meeting in Baltimore on May 5, 2024, Almeida-Porada introduced the first presentation of the scientific symposium “Prospects for Prenatal Gene and Cell Therapy.”

Scientists at Massachusetts General Hospital have linked the risk of heart failure during pregnancy and senescence proteins produced by placental aging, which could clarify how peripartum cardiomyopathy (PPCM) is triggered and opens the door to the development of cardiac function therapies in late pregnancy.

In what represents its first patenting, Sonura LLC has been issued with a patent for an aural device designed to protect the hearing of infants in a neonatal intensive care unit (NICU) by active filtering, while, at the same time, providing aural stimulation for neurological development.

Organoids are 3D models created from human stem cells and resemble fetal tissues. In an article published in Nature Medicine on March 4, 2024, researchers from University College London provided details on the possibility of generating organoids from epithelial cells collected from amniotic fluid without terminating the pregnancy.

Neurenati Therapeutics has closed its seed funding round, securing CA$1.2 million (US$884,000) to advance development of therapies for various rare diseases, including pediatric conditions.

Researchers from Texas A&M University System have detailed the development and characterization of a novel pediatric rat model of organophosphate (OP)-induced status epilepticus (SE).