HOUSTON (AUGUST 19, 2025) – Texas Children’s is pleased to announce that a three-year-old girl has been successfully treated with the first-ever FDA-approved gene therapy treatment for AADC deficiency.    Aromatic l-amino acid decarboxylase (AADC) deficiency is an extremely rare, inherited neurological disorder that prevents the brain from producing dopamine and serotonin — essential chemicals for controlling movement, mood and basic nervous system functions. The literature reports approximately 350 people with this condition worldwide. Historically, there was no cure or approved treatment for AADC, and the shortened life expectancy was estimated between ...

A new vaccine for pneumococcal disease will be tested under an international trial aiming to provide greater protection to babies against the common infection that causes pneumonia, sinusitis and meningitis. The Murdoch Children’s Research Institute (MCRI) led study is evaluating a promising vaccine developed to protect against 21 strains of pneumococcus, up from the current 13 strains included in the National Immunisation Program (NIP). The Melbourne arm of the randomised controlled trial is recruiting 50 families with heathy two month olds who haven’t had ...

Heat waves — prolonged periods of abnormally hot weather — influence egg prices, energy bills and even public transit. And they’re becoming more common as temperatures increase. In a new study, UIC researchers report that heat waves across Africa are hotter, longer and more frequent today than 40 years ago, mainly due to increased greenhouse gas and black carbon emissions from burning fossil fuels. Understanding heat waves’ origins and effects can help African countries predict them and ...

Mental health providers are trained to guide others through trauma, yet their own exposure to clients’ suffering can take a significant toll. Studies show that between 40% and 85% of providers experience compassion fatigue or secondary traumatic stress – key signs of reduced professional quality of life. These stressors are often ongoing and layered, and when combined with limited coping tools and a sense that they have little control over their circumstances, the impact can be even greater. Researchers from Florida Atlantic University examined how different coping styles affect the relationship between a provider’s sense of control over ...

Background and Aims Chronic hepatitis B virus (HBV)-infected patients may exhibit liver fibrosis and other pathological changes despite normal alanine aminotransferase (ALT). This study aimed to assess the efficacy and safety of tenofovir amibufenamide (TMF) in chronic HBV-infected patients with normal ALT levels. Methods The ongoing PROMOTE study (NCT05797714) is the first prospective, multicenter, randomized, open-label, blank-controlled clinical trial involving chronic HBV-infected patients with normal ALT levels. Participants were randomized in a 1:1 ratio to receive ...

Automated insulin delivery (AID) systems such as the UVA Health-developed artificial pancreas could help more type 1 diabetes patients if the devices become fully automated, according to a new review of the technology. Even as the artificial pancreas and other AID systems have helped millions of people with type 1 diabetes better manage their blood sugar and improve their overall health, there are still limitations to overcome, according to the review from University of Virginia Center for Diabetes Technology scientists and their colleagues. These limitations include: AID systems are not yet fully ...

West Nile virus (WNV) has been the dominant cause of mosquito-borne illness in the United States since its introduction into North America in 1999. There are no vaccines nor medications to prevent or treat illness in people, so surveillance, prevention, and control remain the best options to protect the public. Mosquito surveillance for WNV is a central component of the public health response, but this approach is labor intensive and limited by practical constraints on the number of locations that can be sampled. To address this limitation, Joseph McMillan and colleagues developed a validated machine learning model that uses freely ...

Sub-Saharan Africa currently has the highest infant mortality rate in the world, with 27 babies out of every 1,000 live births dying in their first month. As the climate warms, pregnant women in the region are increasingly exposed to extreme heat, which can cause reduced placental blood flow and dehydration, potentially affecting fetal development. In addition, extreme heat can lead to the proliferation of pathogenic bacteria in the environment, and can make it  difficult for women to travel to prenatal care appointments. Jiafu An and colleagues sought to determine whether in-utero exposure to extreme ...

MINNEAPOLIS / ST. PAUL (08/19/2025) — Researchers at the University of Minnesota Twin Cities have made a groundbreaking discovery by observing and analyzing the first new type of plasma wave in Jupiter’s aurora. This research helps us understand “alien aurora” on other planets, which in turn teaches us more about how Earth’s magnetic field protects us from the sun’s harmful radiation.  The research is published in Physical Review Letters, a peer-reviewed, multidisciplinary, high-impact scientific journal. The observation is based on data from NASA’s Juno spacecraft, which made a historic low ...

Stroke remains one of the leading causes of long-term disability worldwide, affecting millions each year. Among its most debilitating consequences is gait impairment, which affects over 80% of stroke survivors. Impaired walking not only reduces independence but also limits participation in daily and social activities, significantly diminishing the quality of life. While some patients regain partial ambulatory function, walking speed and coordination often remain impaired. Alarmingly, up to 22% of survivors never regain the ability to walk and are ...

Cerebrovascular diseases such as atherosclerosis and stroke remain a major cause of morbidity and mortality worldwide. A common feature of these diseases is vascular stenosis, i.e., the narrowing of blood vessels, which disrupts normal blood flow and contributes to chronic inflammation in the vessel wall. Endothelial cells lining the vasculature play a key role in sensing shear stress from blood flow and responding to disturbed hemodynamics by expressing pro-inflammatory molecules. However, studying this phenomenon in vivo is challenging due to the complexity ...

The tongue contains numerous taste buds—tiny sensory organs responsible for detecting taste. Taste buds consist of specialized cells that translate chemical stimuli into neural signals. Among them, type II cells, which respond to sweet, umami, and bitter stimuli, utilize channel-based signal transmission. In contrast, type III cells are believed to mediate sour signals via synaptic vesicle release. While the signaling mechanisms of type II cells are well-characterized, the process of vesicular synaptic transmission in type III cells is poorly understood.   Synaptic signal transmission relies on the SNARE complex, ...

The International Progressive MS Alliance announces the launch of its MS Clinical and Imaging Data Resource (CIDR), which provides the academic and research community access to anonymized and harmonized MRI and clinical data from thousands of people living with MS who participated in one of many clinical trials. The Alliance, in conjunction with McGill University in Montréal, Canada, and four industry partners – Biogen, Novartis, Roche and Sanofi - is making this resource available exclusively to the MS research community to help accelerate ...

Osaka, Japan－Researchers at The University of Osaka have discovered a new type of chiral symmetry breaking (CSB) in an organic crystalline compound. This phenomenon, involving a solid-state structural transition from an achiral to a chiral crystal, represents a significant advance in our understanding of chirality and offers a simplified model to study the origin of homochirality. This transformation also activates circularly polarized luminescence, enabling new optical materials with tunable light properties. Chirality, or "handedness," is a fundamental property of objects, from galaxies to molecules, and plays a crucial role in biological ...

A newly developed system transforms human waste into a powerful tool for profitable and sustainable energy and agriculture in resource-limited regions. The prototype, outlined in a Stanford-led study published Aug 19 in Nature Water, recovers a valuable fertilizer from urine, using solar energy that can also provide power for other uses. In the process, the system provides essential sanitation, making wastewater safer to discharge or reuse for irrigation. “This project is about turning a waste problem into a resource opportunity,” said study ...

Aging is particularly harsh on the hippocampus — the brain region responsible for learning and memory. Now, researchers at UC San Francisco have identified a protein that’s at the center of this decline.  They looked at how the genes and proteins in the hippocampus changed over time in mice and found just one that differed between old and young animals. It’s called FTL1.  Old mice had more FTL1, as well as fewer connections between brain cells in the hippocampus and diminished cognitive abilities.  When the researchers artificially increased FTL1 levels in young mice, their ...

LA JOLLA (August 19, 2025)—Nearly everything you know about plants was first discovered in a plant you’ve likely never heard of. Arabidopsis thaliana, also known as thale cress, is a small, flowering weed that has shaped much of plant biology as we know it. Serving as the representative plant species in most plant research across the last half century, Arabidopsis has taught us how plants respond to light, which hormones control plant behavior, and why some plants grow long, deep roots ...

CAMBRIDGE, Cambridgeshire, UNITED KINGDOM, 19 August 2025 -- In a revealing Genomic Press Interview published today in Brain Medicine, Dr. David Rubinsztein shares the remarkable journey that led him to discover how cells naturally clear toxic proteins that cause devastating neurodegenerative diseases. The comprehensive interview unveils both the scientific breakthroughs and personal philosophy that have positioned autophagy modulation at the forefront of therapeutic innovation for conditions affecting millions worldwide. From Cape Town Curiosity to Cambridge Discovery Dr. Rubinsztein traces his scientific awakening to childhood in South Africa, ...

University of Warwick researchers have built a new diamond-based magnetic field sensor that could be used to better find tumours through tracing magnetic fluid injected in the body.  A cancer diagnosis is most problematic when cells from the tumour have metastasised (spread) to other organs. This most often occurs through the lymph nodes and the lymphatic draining system. Tests to find whether cancer cells are lodged in the lymph nodes are the gold standard for detecting metastasis and directing the course of treatment. Published in Physical Review Applied, Warwick researchers report they have built a ...

Supernovae appear to our eyes—and to astronomical instruments—as brilliant flashes that flare up in the sky without warning, in places where nothing was visible just moments before. The flash is caused by the colossal explosion of a star. Because supernovae are sudden and unpredictable, they have long been difficult to study, but today, thanks to extensive, continuous, high-cadence sky surveys, astronomers can discover new ones almost daily. It is crucial, however, to develop protocols and methods that detect them promptly; ...

New research finds that Marine Protected Areas can boost the recovery of globally important kelp forests following marine heatwaves. The findings are published in the British Ecological Society’s Journal of Applied Ecology. Using four decades of satellite images, University of California, Los Angeles (UCLA) researchers have looked at impacts Marine Protected Areas (MPAs) are having on kelp forests along the coast of California. They found that although the overall effect of MPAs on kelp forest cover was modest, the benefits ...

Oil spills and oily industrial wastewater are a nightmare for factories, the environment, and public health. Separating oil from water might sound simple, but in reality it's one of the toughest jobs in wastewater treatment—especially when the mixture contains oils of different densities, tiny droplets, or sticky contaminants. Traditional membrane filters often clog, slow down, and lose efficiency over time. In International Journal of Extreme Manufacturing, a research team has developed an organic ...

Osaka, Japan – Life as we know it is based on organic molecules. In these molecules, carbon and hydrogen atoms are linked into a fascinating array of structures, such as chains or rings. One special class of organic molecules, hetero[8]circulenes, can behave in interesting ways because of their ring of eight atoms, and have many applications, including electronic devices responsible for controlling and detecting light. However, creating these molecules through planned chemical reactions, or the synthetic route, ...

It’s often mistaken for a heart attack, but Takotsubo cardiomyopathy – previously known as Broken Heart syndrome – is a serious and sometimes fatal heart condition increasingly reported in intensive care units (ICUs). Yet without a clear clinical pathway in ICUs, it’s often missed, putting critically ill patients at risk.   New research from the University of South Australia shows that using electrocardiogram (ECG) patterns and blood markers could provide an early warning system for Takotsubo Syndrome in ICU patients.   The review highlights how critical care nurses with advanced ECG skills can play a key role in recognising early signs of the condition ...

One of the stranger forms of life on our planet is the tiny, torpedo-shaped chaetognath, which roams the seas on the hunt for small crustaceans. These predators are named after the chitinous grasping spines surrounding their mouth (Greek: “chaite”, bristle, and “gnathos”, jaw), and are also known as arrow worms. Despite their ubiquity in the world’s oceans, the evolutionary origin of this unique lifeform has long baffled biologists – Charles Darwin himself noted their “obscurity of affinities” in 1844. Notably, the worm has characteristics of both protostomes, which include ...