Schizophrenia (SZ), bipolar disorder (BP), major depression (MDD) and autism spectrum disorder (ASD) are serious mental illnesses (SMIs) that affect a significant proportion of the worldwide population. Large genome-wide association studies have pointed to overlapping genetics including both common and rare variants as cause of these SMIs. A recent study published on June 16, 2026, in Genomic Psychiatry has shed some light regarding the etiology of SMIs.
Autism spectrum disorder (ASD), developmental epileptic encephalopathies and other neurodevelopmental disorders are driven by the disruption of genes regulating neuronal proliferation, differentiation and synaptic maturation. Researchers from Shanghai Jiao Tong University School of Medicine generated Csnk2b haploinsufficient (Csnk2b+/-) mice mimicking the most relevant disease features to investigate the effects of reduced gene dosage.
Naturecell Co. Ltd.’s U.S. subsidiary, Naturecell America, has received IND clearance from the FDA for Astrostem-AU, an autologous adipose-derived mesenchymal stem cell therapy for adults with autism spectrum disorder (ASD).
Marvel Biosciences Corp., together with its wholly owned subsidiary Marvel Biotechnology Inc., has announced nondilutive funding from the Alberta Innovates CarE (AICE) market access program to support progression into the clinic and phase I testing of lead compound, MB-204. MB-204, a fluorinated derivative version of istradefylline, offers potential as a new approach totreating autism spectrum disorders and related conditions, addressing socio-behavioral symptoms through a differentiated mechanism.
Fast on the heels of the U.S. FDA’s go-ahead regarding the expanded use of Wellcovorin (leucovorin) tablets for cerebral folate deficiency in adult and pediatric patients with a confirmed variant in the folate receptor 1 gene – some of whom bear “autistic features” as part of their condition – the American Academy of Pediatrics advised against giving the drug to children with autism spectrum disorder.
The SCN2A Foundation has entered into a research collaboration with Unravel Biosciences Inc. to advance preclinical research for SCN2A-related disorders caused by loss-of-function mutations, a subset of SCN2A conditions driven by insufficient functional protein.
A new systematic review and meta-analysis of studies looking at a possible association between Tylenol (acetaminophen) use in pregnancy and neurodevelopmental disorders in children concludes there is no evidence of any meaningful risk. The study was carried out by researchers in the U.K., Italy, Sweden and Norway, in response to U.S. Secretary of Health and Human Services Robert Kennedy saying last September that there is a link. Unless medically necessary, it is “irresponsible” to take Tylenol during pregnancy, Kennedy said.
Changes to a U.S. CDC website regarding autism and vaccines has sparked a backlash from numerous scientific and other groups, placing HHS Secretary Robert F. Kennedy Jr. (RFK) in the spotlight once again for appearing to break promises made earlier this year to secure his nomination.
Changes to a U.S. CDC website regarding autism and vaccines has sparked a backlash from numerous scientific and other groups, placing HHS Secretary Robert F. Kennedy Jr. (RFK) in the spotlight once again for appearing to break promises made earlier this year to secure his nomination.
Some 1 million people around the world suffer severe neurological problems, such as epilepsy, motor impairment and cognitive dysfunction, because they express insufficient SynGAP1, a GTPase-activating protein that operates postsynaptically. Current therapies can mitigate symptoms but not cure the underlying disease. Researchers at the Allen Institute and collaborators have demonstrated a potential genetic cure for SynGAP1 deficiency.
