Medtronic plc has agreed to buy Scientia Vascular Inc. for $550 million, as it makes good on its promise to embark on more strategic acquisitions this year. The acquisition will bolster its neurovascular business as it adds a portfolio of guidewires and catheters, which uses Scientia’s microfabrication technology to simplify complex neurovascular procedures.

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.

If one could sweep the brain clean and send the toxic substances that drive neurodegeneration to the recycling bin, perhaps one could treat Alzheimer’s disease. Researchers at the Chinese Academy of Sciences propose a new therapeutic strategy that uses synthetic peptides that bind to amyloid-β (Aβ) and direct it toward lysosomes. In addition, researchers at the Washington University School of Medicine in St. Louis have genetically modified astrocytes in vivo to express chimeric antigen receptors (CARs) that recognize and phagocytose Aβ plaques.

The regulatory clouds that have been darkening the U.S. FDA landscape of late for Uniqure NV’s gene therapy AMT-130 in Huntington’s disease may be parting a bit with the announced departure of Vinay Prasad as director of the agency’s CBER at the end of April.

Any lingering disappointment in the delay for Xenon Pharmaceuticals Inc.’s readout of the phase III X-Tole2 study testing azetukalner in focal onset seizures (FOS) appeared thoroughly extinguished as the company’s K_V7 potassium channel opener yielded better-than-expected data, even besting earlier phase IIb findings and positioning the drug for an NDA submission later this year.

Japan has approved the world’s first therapies derived from induced pluripotent stem cells (iPSCs), marking a major milestone for regenerative medicine and, potentially, a turning point in treating Parkinson’s disease.

Researchers at INSERM and collaborators have identified hypothalamic tanycytes as mediators of tau clearance and shown that their structural and genetic disruption may drive Alzheimer’s disease pathology.

Science Corp. raised $230 million in an oversubscribed series C financing round to commercialize its Prima brain-computer interface (BCI) retinal implant and to advance other pipeline programs into the clinic. The investment brings the company’s total funding, since founding in 2021, to approximately $490 million and shows investors growing appetite for BCI technologies.

At the current pace of innovation in the U.S. rare disease space, developing and approving therapies for just half of the 10,000-plus known rare diseases would take more than 160 years, Bradley Campbell, president and CEO of Amicus Therapeutics Inc., recently told the Senate Committee on Aging.

A therapeutic strategy based on alternative splicing of the MECP2 gene could restore protein levels in Rett syndrome, a neurological disorder caused by mutations in that gene. Scientists at Baylor College of Medicine have successfully tested this approach both in vitro in neurons from Rett patients that produce some functional protein, correcting the altered gene expression and improving neuronal functions, and in vivo in mice.