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Crigler-Najjar syndrome: Gene therapy maintains its efficacy four years after treatment

The New England Journal of Medicine has published a study on the long-term efficacy of AAV8-mediated gene therapy (GNT0003) in patients with Crigler-Najjar syndrome, an extremely rare congenital liver disorder affecting approximately 500 people in Europe. It is caused by a deficiency of UGT1A1, an enzyme required for bilirubin metabolism. The accumulation of this substance can lead to irreversible neurological damage and, in the most severe cases, death.

Medical Xpress · 4h ago
Shedeur Sanders’ response to Browns questions sparks doubts over his NFL starting credentials

Shedeur Sanders has faced criticism over his response to questions about his fit in the Cleveland Browns’ offence, with one radio host questioning whether he has what it takes to become an NFL starting quarterback. The rookie is preparing to step in for Deshaun Watson, who entered concussion protocol after reporting symptoms following Cleveland’s 27-6 defeat to the Pittsburgh Steelers.

Yahoo Sports · 12h ago
An anti-aging drug may help protect the brain before Alzheimer's symptoms begin

A surprising new study suggests that an anti-aging drug could help protect people genetically predisposed to Alzheimer's disease. Researchers found that just four weeks of low-dose rapamycin improved brain blood flow in healthy middle-aged adults carrying the high-risk APOE4 gene variant, with women showing the strongest benefits. Because declining blood flow can occur years before Alzheimer's symptoms appear, the discovery raises hopes for preventing or delaying the disease before memory loss begins.

Science Daily · 19h ago
France needs 'more public services', 'salaries are too low', 'schools are in very, very bad shape'

As France confronts another wave of strikes and protests, Gaëlle Martinez, National Secretary of the trade union Solidaires, presents the general strike as more than a confrontation over wages, public spending or the cost of living. Her account reveals a deeper struggle over economic dignity, the future of public services and the democratic right to collective protest. “People just want to live, not survive.” For Martinez, deteriorating schools, insufficient salaries and the dismantling of public services are not isolated policy failures. They are interconnected symptoms of political and economic priorities that, in her assessment, place fiscal restraint above social welfare. Her demand for “a new budget” that is “not based on cuts” is accompanied by a call to “tax the biggest companies, the richest companies where the profits are made.”

France 24 · Nov 5
One gene, two paths: Opposite synaptic defects linked to same intellectual disability

A research team led by Dr. Sheng Nengyin of the Kunming Institute of Zoology (KIZ) of the Chinese Academy of Sciences has reframed ZC4H2-associated rare disorder (ZARD)—a disorder marked by intellectual disability, developmental delays, muscle weakness and other neurological or physical abnormalities—as a "disease spectrum" rather than a condition driven by a single mechanism.

Medical Xpress · Nov 5
Scientists discover mysterious “lonely” brain waves linked to memory loss

Scientists have uncovered a surprising link between Alzheimer’s-related protein buildup, disrupted brain waves during deep sleep, and fading memory. The findings suggest that hidden changes in how the brain processes memories overnight may contribute to age-related memory loss long before Alzheimer’s symptoms appear.

Science Daily · Nov 5
Microglia drive excessive iron transfer in Parkinson's disease models

Parkinson's disease (PD) is a neurodegenerative condition associated with worsening tremors, slowed movements, changes in walking and speech, muscle rigidity and postural instability. Many of its symptoms are associated with a progressive loss of dopamine-producing neurons in a brain region called the substantia nigra pars compacta.

Medical Xpress · Nov 5
Rapid tau spread may explain why midlife Alzheimer's progresses more rapidly

Patients with early-onset Alzheimer's disease, whose symptoms start before age 65, have more extensive deposits of tau, a hallmark protein of the condition, compared with patients whose symptoms start at 65-plus, a new study found. What's more, tau accumulates at more than twice the rate in younger compared to older patients and spreads widely throughout the brain.

Medical Xpress · Nov 4
At-home exercise may help teens bounce back faster from concussion

One doesn't have to step onto a football field to get a concussion; it can happen anywhere. A fall during an everyday activity or a minor car accident can deliver the jolt to the head that causes one. While headaches, memory problems, disrupted sleep and difficulty concentrating usually fade without a trace, up to 30% of people continue to experience these symptoms for years.

Medical Xpress · Nov 3
Are the UK and Canada next? Are we? Lessons from Europe’s smoldering streets

The smoke rising from Marseille, the smashed storefronts of Frankfurt, and the military patrols deployed to the streets of Milan over the weekend are not isolated incidents of youthful misbehavior. They are the symptoms of a dying status quo. What began as scattered student marches has metastasized into a continent-wide explosion of civil unrest. As […]

Washington Examiner · Nov 3
Human dengue trial reveals how immune memory can help—or backfire

Spread through the bite of Aedes mosquitoes, dengue is a viral infection that can cause anything from mild fever, flu-like symptoms and joint pain to a severe form known as dengue hemorrhagic fever. What makes dengue particularly unusual and dangerous is that a second infection with a different strain, or serotype, can sometimes lead to more severe disease rather than stronger immunity.

Medical Xpress · Nov 2
Flu Season Kicks off Early: Is it Too Late to Get the Vaccine?

Flu activity is higher than usual for this time of year, according to the CDC, so NBC News medical contributor, Dr. Akshay Syal, joins TODAY with tips to stay healthy. He discusses why there’s an early uptick in numbers, how to protect against the strain that is circulating, and how to manage your symptoms if you do get the flu.

NBC News · Nov 2
‘We both know how we feel about death and dying’: Rhys Ifans and Laura Linney on their brutally honest Parkinson’s drama

Written by Vicar of Dibley co-creator Paul Mayhew-Archer, who has the disease, Onwards and Sideways sees the actors play a couple who bond over their diagnoses – a devastating subject but laced with humour too, they say Ahead of production on their first job together, Rhys Ifans and Laura Linney didn’t undergo the standard “chemistry read” to make sure they got on. Which is remarkable, given what their characters would have to do to each other in Onwards and Sideways. In the Norfolk-set BBC film directed by John Madden (Shakespeare in Love), Ifans would have to nearly throttle Linney while in the grip of night terrors, she’d have to collapse in his arms in a weeping mess having bankrupted herself with a gambling addiction; both situations arising from the neurological condition afflicting their characters: Parkinson’s disease. Continue reading...

The Guardian · Nov 2
The UK’s legal cannabis market is booming – so why can’t this talented artist get any to treat his epilepsy? | Kojo Koram

Companies have rushed to supply the drug as a wellness treatment, but patients with serious illnesses aren’t able to get NHS prescriptions In 2022, a prodigiously talented young artist named Louis Petit was preparing to return to the UK, having been effectively exiled in the Netherlands as a teenager so he could access the medical cannabis he needed to control his epilepsy. Six years earlier, Louis, then 12, had been diagnosed with a rare form of the neurological disease when medical cannabis was still illegal in the UK. But in Rotterdam, where it was legal, doctors helped him discover that a medical cannabis oil called Bedrolite, in combination with the right mix of anti-epileptic treatment, could slow and eventually stop his seizures. This was something his neurologists in the UK had previously considered impossible. Dr Kojo Koram is professor of law and political economy at Loughborough University and the author of The Next Fix: The Winners and Losers in the Future of Drugs Continue reading...

The Guardian · Nov 1
Treatment with norrin helps maintain healthy blood vessels and preserve vision in diabetic rats

Millions of people in the U.S. are affected by diabetic retinopathy, a complication of diabetes that affects the eyes. The condition is caused by damage to the blood vessels in the retina, the light-sensitive tissue at the back of the eye. At first, diabetic retinopathy might not cause any symptoms, or the problems might be mild. However, if the underlying diabetes remains poorly managed, it can lead to blindness.

Medical Xpress · Oct 31