The primary-endpoint win by Sensorion SA in a phase II proof-of-concept study with SENS-401 (arazasetron) in hearing loss made public March 11 brought renewed interest in the space, where a number of players are advancing gene therapies. The story marches on, with Montpellier, France-based Sensorion due to discuss the product July 13 at the International Conference on Cochlear Implants and Other Implantable Technologies in Vancouver, British Columbia.
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.
The word “niche” implies a specialized environment. But to Fiona Doetsch, the stem cell niche is anything but. For brain stem cells, “the whole organism is the niche,” Doetsch told the audience at the third plenary session of the International Society for Stem Cell Research (ISSCR) annual meeting in Hamburg this week. It’s a surprising idea at first, given the brain’s protection from many circulating substances via a series of barriers, including the blood-brain barrier and the blood-cerebrospinal fluid barrier.
Nonalcoholic steatohepatitis was renamed, for the first time in 34 years, to metabolic dysfunction associated steatohepatitis (MASH), but a name change is far from being the biggest development in the field, according to experts at Bioplus Interphex (BIX) Korea 2024.
Ependymoma is a brain cancer that occurs during childhood and is usually resistant to cytotoxic therapy. Molecular profiling has helped in better understanding the mechanisms behind the disease and has unveiled EPH receptor B2 (EPHB2) as a critical player in driving disease.
The industry is looking, with renewed hope, to the “promise” of messenger RNA (mRNA) therapeutics for a wide range of diseases beyond COVID-19, and not only in vaccine form but also for gene and cell therapies.
Researchers from the University of California San Francisco and Northwestern University recently presented findings from a study that aimed to evaluate the role of lin-28 homolog B (LIN28B), an RNA binding protein expressed in a variety of cancers, in diffuse midline glioma (DMG).
Many patients with amyotrophic lateral sclerosis (ALS) or frontotemporal dementia (FTD) harbor the (G4C2)n pathogenic repeat expansion in the C9orf72 gene, which leads to aggregating dipeptide proteins, mainly poly-glycine-alanine (poly-GA).
Recent studies have highlighted the potential role of the small GTPase Rac1 as new therapeutic target in SHH-medulloblastoma due to its regulation of Hedgehog signaling via the GLI1 and GLI2 transcription factors.