Researchers led by Kyle Loh at Stanford Medicine discovered that the forebrain and hindbrain arise from distinct progenitor cells rather than a single common origin, contradicting decades of neuroscience theory. The forebrain governs higher cognition like language and abstract thought, while the hindbrain controls vital functions such as heartbeat and breathing. The study, published in Nature Neuroscience, was co-led by graduate students Carolyn Dundes and Rayyan Jokhai.
A man with a slow-progressing, genetically rare form of ALS became the first person to receive an antisense oligonucleotide therapy designed specifically to target his causal mutation. A year later, he reported improved symptoms and remains able to work as a physician, according to results published in Med.
Investors are reportedly re-entering rare-disease drug stocks after the departure of stricter FDA regulators under the Trump administration. The shift in agency leadership has reduced concerns about tougher approval hurdles that had previously discouraged investment in this biotech segment.
Researchers analyzed human brain tissue samples from people with and without Alzheimer's disease and identified more than 1,000 microproteins that had previously gone undetected. Dozens of these small proteins showed different expression levels in Alzheimer's-affected brains compared with healthy ones. Separately, a mouse study found that immune cells activated by gut infections can travel to the brain's protective membrane and remain there as a lasting defensive memory.
Researchers propose that Alzheimer's disease progresses through distinct molecular and cellular phases marked by biological thresholds, rather than a simple straight-line path from amyloid buildup to Tau pathology to neurodegeneration. At these thresholds, cells lose homeostatic balance and shift behavior, with these altered states spreading unevenly across brain tissue and eventually reaching regions critical to cognition. A key threshold identified is when amyloid triggers Tau phosphorylation, marking the transition from amyloid-driven tissue states to ones dominated by neuronal stress responses.
Scientists at the Salk Institute and collaborators combined multiple detection techniques to identify over 4,300 previously unrecognized 'microproteins' in postmortem brain tissue from the dorsolateral prefrontal cortex, comparing samples from people with and without Alzheimer's disease. Dozens of these tiny proteins, each under 150 amino acids, showed altered levels in Alzheimer's patients, and the findings were published in Nature Aging on September 14.
The Rat and Mouse Gazette published detailed guidance for owners caring for severely ill or aging rats, covering isolation from cage mates, warmth, bedding, and hygiene. It recommends moving very sick rats to a separate aquarium with soft bedding like old T-shirts, using a wrapped heating pad for warmth, and gently wiping down rats too weak to groom themselves.
Researchers examined the physics of toilet flushing and found that the aerosol plume generated can rise rapidly and disperse droplets to roughly face height within seconds. They identified specific factors, including flush design and water pressure, that increase how far and how high these microbial-laden particles travel.
Google DeepMind released AlphaGenome Atlas, a genome-wide dataset predicting the functional consequences of nearly every possible single-letter DNA change. Academic partners, including researchers from the Broad Institute working with the GREGoR Consortium, used the tool's AVI scoring system to identify a previously overlooked variant in the DNM1 gene linked to epileptic encephalopathy, showing it creates a faulty splice site that abnormally extends the resulting protein. The prediction was later confirmed through lab experiments.
A case report documents an instance of southern tick-associated rash illness (STARI) diagnosed in New York, a region well outside the disease's traditional southern range. The finding adds to evidence that the condition, which mimics Lyme disease symptoms, is spreading northward as tick populations shift.
Physicians at Houston's Ben Taub Hospital have documented a cluster of previously undiagnosed kidney failure cases among young, otherwise healthy immigrant laborers from Mexico and Central America. Internist Ricardo Nuila suspects these cases resemble chronic kidney disease of unknown origin (CKDu), a condition long seen among sugarcane workers in El Salvador and other Central American countries but never before confirmed in the United States. The patients, many employed in physically demanding outdoor jobs like roofing, painting, and road paving, now require dialysis several times a week to survive.
A phase of clinical testing involving sixteen patients with multiple sclerosis and related autoimmune disorders showed symptom improvement after researchers used a modified virus to instruct T cells inside the body to produce chimeric antigen receptors, targeting the B cells responsible for autoimmune attacks. The findings, published this week in The New England Journal of Medicine, mark one of the first demonstrations that CAR-T therapy can be generated directly inside a patient rather than engineered in a lab and reinfused.
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