Investigators at the Institute for Research in Biomedicine (IRB Barcelona) have unraveled how and why the absence of a neuronal microexon in cytoplasmic polyadenylation element-binding 4 (CPEB4) gives rise to autism. In 2018, investigators from IRB, co-led by Raúl Méndez, identified the overt correlation between defects in CPEB4 and the onset of autism. However, the previous work did not provide the molecular mechanism explaining the correlation.
By the end of November 2024, the BioWorld Neurological Diseases Index had dropped 13.4%, deepening its decline of 9.4% at the end of August. Meanwhile, the Dow Jones Industrial Average surged 19.16% in the first 11 months of the year, building on a 10.81% gain reported at the end of October. The Nasdaq Biotechnology Index posted a year-to-date increase of 6.27% through November, retreating from its peak gain of 11.7% in August.
News that Trevi Therapeutics Inc. got the size just right of the phase IIb Coral trial with Haduvio (oral nalbuphine extended release) led the company’s shares (NASDAQ:TRVI) to close Dec. 12 at $3.60, up $1.11, or 45%, after trading as high as $4.60.
AC Immune SA has divulged compounds acting as α-synuclein (SNCA) aggregation inhibitors reported to be useful for the treatment of multiple system atrophy (MSA), Parkinson’s dementia, Lewy body dementia, Alzheimer’s disease and Parkinson’s disease, among others.
Researchers from State University of New Jersey (Rutgers) presented preclinical data for PW-507, a sigma-1 receptor antagonist being evaluated for the treatment of binge eating disorder.
Pathogenic variants in the KCNT1 gene, which encodes potassium channel subfamily T member 1, cause a severe childhood developmental epileptic encephalopathy.
CNS specialist Noema Pharma AG has added a further $44 million to its series B, bringing the total for the round to $147 million. The new financing bolsters the balance sheet as the Basel, Switzerland-based company progresses four phase II trials, with key data readouts expected in 2025.
Epileptic encephalopathy due to mutations in the STXBP1 gene, also known as genetic epilepsy, is a rare disease characterized by intellectual disability, speech and motor impairment and behavioral issues, among others, that affects 1 in every 30,000 newborns and which has no approved therapies to date.
Researchers from Maplight Therapeutics Inc. presented preclinical data for the investigational muscarinic M1/M4 receptor agonist ML-007, being evaluated for the treatment of neuropsychiatric disorders, such as schizophrenia and Alzheimer’s disease (AD). The activity of ML-007 was compared to that of another muscarinic M1/M4 agonist, xanomeline.
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