On its May 10 PDUFA date, the U.S. FDA approved serotonin-dopamine modulator Rexulti (brexpiprazole) from Otsuka Pharmaceutical Co. Ltd. and H. Lundbeck A/S for agitation in Alzheimer’s dementia, marking the first approved treatment for the indication.
A new mouse model of an inherited form of dystonia has shown the spinal cord is the driver of the condition, overturning previous understanding that the movement disorder is caused by disruption of neural circuits in the brain. The connection was demonstrated by selectively deleting torsin family 1 member A (TOR1A), the gene that causes dystonia, in the neurons of the spinal cord only.
The aqueous supernatants resulting from ultracentrifugation of brain samples from patients with Alzheimer’s disease (AD) contain aggregates so far described as soluble oligomers of amyloid-β protein (Aβ), which are responsible for the neurotoxicity underlying AD and thus considered targets to watch in this devastating condition. Now, a group of scientists from Harvard Medical School have determined that these aggregates are in fact insoluble diffusible fibrils with the same atomic structure as plaque fibrils.
University of Sydney spinout Kinoxis Therapeutics Pty announced a partnership and licensing agreement with Boehringer Ingelheim GmbH to develop first-in-class oxytocin-targeting precision psychiatry treatments to improve the quality of life of people living with neuropsychiatric disorders.
Compounds reported to be useful for the treatment of myotonic 1, myotonic 2 and muscular dystrophy have been described in a City University of New York patent.
Replacing the damaged cell population in neurodegenerative diseases provides a treatment approach. However, interventions that protect host cells could offer new treatment possibilities for neurodegenerative diseases, such as Parkinson’s and Alzheimer’s diseases and amyotrophic lateral sclerosis (ALS).
New optically active benzodiazepines have been reported in a recent Kyonggi University patent as potentially useful for the treatment of diabetic neuropathy.
Suzhou Genassist Therapeutic Co. Ltd. has announced its pre-IND application of its first base-editing product, GEN-6050, and acceptance by the FDA. GEN-6050 is an in vivo base-editing drug that targets exon 50 skipping in the Duchenne muscular dystrophy (DMD) gene.
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