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.
Merck Sharp & Dohme LLC (MSD) has prepared and tested new positron emission tomography (PET) agents for binding and imaging α-synuclein (SNCA) for the diagnosis of Parkinson’s disease.
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.
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 (AD) pathology. AD is characterized by the buildup of extracellular amyloid-β plaques and intracellular tau tangles, protein aggregates that disrupt neuronal function and drive neurodegeneration.
Neurosterix Pharma Sarl has divulged 3-cyclopropylpyrazole derivatives acting as muscarinic M4 receptor positive allosteric modulators. They are reported to be useful for the treatment of psychiatric and neurological disorders.
Targeting the interaction between the C-terminal domain of the GluA2 subunit of AMPAR and brefeldin-resistant Arf-GEF 2 (BRAG2), a guanine nucleotide exchange factor for the small GTPase Arf6, presents a potential therapeutic approach for acute ischemic stroke.
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.
Vanda Pharmaceuticals Inc. will get to take its argument for twice-rejected jet lag disorder drug Hetlioz (tasimelteon) before the U.S. FDA in a formal evidentiary public hearing, a rare move by the agency that the firm claims underscores the “gravity of the legal and scientific issues” it has raised.
A lot of distance lies between talking regulatory flexibility and actually being flexible. That message was driven home again after Uniqure NV disclosed in its latest earnings report March 2 that the U.S. FDA wants a sham-controlled study before it will consider approval of the company’s gene therapy AMT-130 in Huntington’s, a rare disease currently affecting about 41,000 people in the U.S.
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