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An experimental treatment happens to be the solution to delay Alzheimer's symptoms in some people. These people are the ones who are genetically destined to get the disease in their 40s or 50s. These new findings form ongoing research has now been caught up in Trump administration funding delas. The early results of the study has been published on Wednesday and the participants too are worried that politics could cut their access to a possible lifeline.
One of the participants had said, "It is still a study but it has given me an extension to my life that I never banked on having." The participant is named Jake Henrichs, form New York City, who is 50 years old. He is one of them to be treated in that study for more than a decade now and has remained symptom-free despite inheriting an Alzheimer's-causing gene that had killed his father and brother around the same age.
Two drugs which can modestly slow down early-stage Alzheimer's are sold in the United States. These drugs clear the brain of one of its hallmarks, a sticky gunk-like part called the amyloid. However, there have not been any hints that removing amyloid far earlier, way many years before the first symptoms appear, may postpone the disease.
The research is led by Washington University in St Louis, which involved families that passed down rare gene mutation as participants. This meant it was almost guaranteed that they will develop symptoms at the same age their affected relatives did.
The new findings is based on a subset of 22 participants who received amyloid-removing drugs the longest, on average eight years. Long-term amyloid removal cut in half their risk of symptom onset. The study is published in the journal Lancet Neurology.
Washington University's Dr Randall Bateman, who directs the Dominantly Inherited Alzheimer's Network of studies involving families with these rare genes says, "What we want to determine over the next five years is how strong is the protection. Will they ever get the symptoms of Alzheimer’s disease if we keep treating them?”
The researchers before though did not know what exactly caused Alzheimer's which affects nearly 7 million Americans, most of them in their later life. However, it is clear that these silent changes occur in the brain at least two decades before the first symptom shows up. The big contributor. At some point amyloid buildup can trigger a protein named tau that then starts to kill neurons, which can lead to cognitive decline.
Researchers are now thus studying the Tau-fighting drugs and are looking into other factors, like inflammation, brain's immune cells and certain virus.
The National Institute of Health (NIH) has expanded its focus as researchers have found more reasons for Alzheimer's. In 2013, the NIH's National Institute on Aging funded 14 trials of possible Alzheimer's drugs over a third targeting amyloid. By last fall, there were 68 drugs and 18% of them target amyloid. However, there are scientists too who think that amyloid is not everything and their is way more in the brain tissue, immune cells, and more which can be studied.
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The US Food and Drug Administration (FDA) has expanded the approval of Vertex Pharmaceuticals' Casgevy (exagamglogene autotemcel), making it the first gene therapy approved for children as young as 2 years with sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).
Earlier, the one-time CRISPR-based gene-editing therapy was approved only for patients aged 12 years and older. The expanded label is expected to make approximately 5,500 additional children in the US eligible for treatment, the company said.
The FDA said earlier intervention could help prevent irreversible organ damage and improve long-term health outcomes.
“With today’s decision, pediatric patients as young as 2 years of age can now access a critical additional treatment option to treat these debilitating, life-threatening diseases,” said Karim Mikhail, Acting Director of the FDA's Center for Biologics Evaluation and Research (CBER).
"Making this therapy available to younger patients opens a critical window for intervention and gives these children a meaningful chance at a healthier future," added Megha Kaushal, Acting Deputy Director of the FDA's Office of Therapeutic Products.
Also read: Scientific Breakthrough: US Scientists Create First Synthetic Cell That Eats and Reproduces
The expanded approval is backed by clinical trial data in children aged 5 to under 12 years.
For sickle cell disease:
For transfusion-dependent beta thalassemia:
Casgevy is a one-time gene-editing therapy that uses CRISPR technology to modify a patient's own blood stem cells, enabling the body to produce healthier red blood cells.
For people with sickle cell disease, it aims to prevent painful vaso-occlusive crises. In beta thalassemia, it can eliminate the need for regular blood transfusions in many patients.
“These disorders carry a heavy burden for children and their families, affecting growth, development, and long-term health in profound ways,” said Kaushal, also a pediatric hematologist. “Grounded in the scientific evidence that earlier treatment reduces the risk of lasting end-organ damage, making this therapy available to younger patients opens a critical window for intervention and gives these children a meaningful chance at a healthier future.”
The FDA granted the expanded approval through its Commissioner's National Priority Voucher program, completing the review in 53 days.
Vertex said it has also submitted applications to expand Casgevy's use in the United Kingdom and Saudi Arabia, where regulatory reviews are currently underway.
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A popular snack brand is currently under scrutiny after its multiple products were classified under the US Food and Drug Administration's (FDA) Class I recall. The move came after concerns over its product being contaminated with Salmonella surfaced.
According to the FDA, the recall was initiated in May by Utz Quality Foods LLC after the company found out that a seasoning ingredient containing milk powder, sourced from California Dairies Inc. through a third-party supplier, may have been contaminated with Salmonella.
The recall affects a few varieties of its Zapp's and Dirty’s potato chips, including Zapp's Bayou Blackened Ranch, Big Cheezy, and Salt and Vinegar potato chips, as well as Dirty brand Salt and Vinegar, Maui Onion, and Sour Cream and Onion potato chips.
According to the FDA, Utz said the seasoning batches tested negative before being used in production, but the decision to recall the products was taken as a precautionary measure.
While no illnesses have been reported yet, the FDA's move indicates there is a reasonable probability that consuming these products could result in adverse health consequences and even death.
Also read: Cyclospora Parasite Spreads Across 17 US States: How To Prevent Parasitic Infection
The FDA uses three categories to classify product recalls based on the level of public health risk. A Class I recall is usually reserved for products that pose the highest risk to consumers.
It is issued when there is a reasonable probability that consumption or use of the product will cause serious health problems or death.
Salmonella infection, known as salmonellosis, usually develops between six hours and six days after consuming contaminated food.
Also read: Doctors Find 38 Parasites In UK Woman’s Brain After Her Trip To India; What Is Neurocysticercosis?
Common symptoms of salmonellosis include:
Most healthy people recover within four to seven days without targeted treatment. However, it can become severe when the infection enters the bloodstream. Deemed as a medical emergency, this situation prompts urgent attention.
Those with weakened immunity systems, such as young children, older adults, and women who are expecting are at the greatest risk of developing serious complications from salmonellosis. In rare cases, the infection can become life-threatening.
The FDA has advised consumers not to eat the recalled potato chips. It has also said that anyone who has purchased the affected products should either discard them or return them to the place where they bought them for a refund.
The regulatory body has also said that consumers who experience symptoms of salmonellosis after eating the recalled products, especially those at higher risk for complications, should seek medical care immediately.
Although no infection cases linked to the recalled chips have been reported to date, health officials say the recall is intended to prevent potential cases before they occur.
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Women diagnosed with polyendocrine metabolic ovarian syndrome (PMOS) should receive an annual NHS health review and be diagnosed earlier to reduce their long-term health risks, according to draft guidance released by the UK's National Institute for Health and Care Excellence (NICE).
The guidance, now open for public consultation, recommends yearly reviews to monitor symptoms, treatment and the risk of developing serious conditions such as type 2 diabetes and cardiovascular disease. Earlier diagnosis, NICE says, could help people access timely care and make lifestyle changes before complications develop.
PMOS, until recently known as polycystic ovary syndrome (PCOS), was renamed following an international consensus published in The Lancet. The new name better reflects the condition's endocrine, metabolic and ovarian features.
Also read: PCOS Is Now PMOS: What The Name Change Means For Millions Of Women
NICE recommends that everyone diagnosed with PMOS should be offered an annual review covering:
The guideline also includes recommendations on fertility treatment, stating that eligible patients should be offered IVF in line with existing NHS fertility guidance. It also addresses the management of common PMOS-related conditions, including acne, hirsutism and obstructive sleep apnea.
Given the increased risk of metabolic and cardiovascular disease in people with PMOS, the guidance directs healthcare professionals to relevant NICE recommendations for managing these conditions.
Read More: Teplizumab: UK NHS To Roll Out World-First Drug To Delay Onset Of Type 1 Diabetes
The draft guideline highlights that PMOS remains underdiagnosed despite affecting an estimated one in eight women.
It recommends investigating PMOS in anyone with irregular or absent menstrual cycles alongside signs of excess male hormones, as these are common indicators of the condition.
The guidance covers girls aged over 10, as well as adult women, trans men and non-binary people who are not receiving gender-affirming hormone therapy or surgery.
People with suspected PMOS should be offered blood tests to measure hormone levels and, where appropriate, pelvic ultrasound scans. The guideline also states that PMOS should not be ruled out after menopause and notes that the condition may be more prevalent among women of Black, Asian and mixed ethnic backgrounds.
Marie Anne Ledingham, consultant clinical adviser for women's and reproductive health at NICE, said the guidance is intended to improve consistency of care and ensure people receive appropriate long-term monitoring.
"PMOS is a common but often overlooked condition that can have a major impact on health and well-being. Recommending a simple annual review is an important step towards ensuring people get the ongoing care and monitoring they need," Ledingham said, in a statement.
The draft guidance recognizes that PMOS affects far more than reproductive health. It is associated with an increased risk of depression, anxiety, eating disorders, fatty liver disease, obstructive sleep apnoea, pregnancy complications and fertility problems.
For people planning a pregnancy, NICE recommends advice on achieving and maintaining a healthy weight, balanced nutrition, regular exercise, good sleep and mental wellbeing to improve reproductive outcomes.
The guidance also stresses that eating disorders should not be overlooked or dismissed based solely on a person's weight.
While the draft guideline includes recommendations to manage hirsutism, it does not support routine NHS funding for laser and light-based hair removal therapies. NICE concluded that these treatments are not cost-effective, estimating they could cost the NHS "up to £100 million annually" in England if routinely funded.
The draft guideline is open for consultation until August 11, 2026, with the final NICE guidance on PMOS expected to be published in December 2026.
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