Oral Berotralstat Effective and Safe for HAE Prevention in Young Children: Study

A newly published retrospective cohort study (APeX‑P trial) in Journal of Allergy and Clinical Immunology: In Practice found that oral berotralstat—a once‑daily plasma kallikrein inhibitor—was well tolerated and highly effective in reducing hereditary angioedema (HAE) attack rates in children aged 2–11 years. Among the 29 pediatric participants (median age 8), berotralstat led to early and sustained reductions in attack frequency: whereas the baseline median attack rate was 0.96 per four weeks, most children maintained zero to near‑zero attack rates through week 48. There were no drug‑related serious adverse events, discontinuations, or Grade 3/4 toxicities. Mild common side effects included nasopharyngitis, headache, and upper respiratory infections. pharmacokinetics showed a median Tmax of 3.9 hours and consistent exposure levels, supporting reliable once‑daily dosing. These findings suggest berotralstat could be a game‑changer as a first approved oral prophylactic option for children under 12, a population previously limited to injectable prophylaxis. Given its safety and early efficacy, this therapy offers a promising long‑term management strategy that may improve quality of life and reduce the treatment burden for young HAE patients. Experts note that further real‑world studies and longer follow‑up are needed to confirm durability and generalizability across diverse pediatric groups. 

Reference:
Bernatoniene, J., Bourgoin‑Heck, M., Cancian, M., Yang, W., Hagin, D., Pagnier, A., … Stobiecki, M. (2025). Oral berotralstat prophylaxis in children aged 2–11 years with hereditary angioedema: interim results from the APeX‑P trial. JACI: In Practice. https://doi.org/10.1093/jac/dkaf265

Keywords: berotralstat, hereditary angioedema, pediatric prophylaxis, HAE prevention, oral plasma kallikrein inhibitor, attack reduction, safety in children, APeX‑P trial, quality of life, orladeyo

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Parents of children with health conditions less confident about a positive school year

As the new school year begins, some parents may be feeling more nervous than excited—especially those whose children have different health needs.

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Trauma-focused therapy shows promise for children struggling with PTSD

A specialist form of therapy could offer hope for some of the most vulnerable young PTSD sufferers, according to a University of East Anglia study.

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Higher doses of semaglutide can safely enhance weight loss for adults living with obesity, clinical trials confirm

A higher weekly dose of semaglutide (7.2 mg) can significantly improve weight loss and related health outcomes in adults living with obesity, including those with type 2 diabetes (T2D), according to the results of two large-scale, international phase 3 clinical trials.

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Gestational diabetes linked to cognitive decline in mothers, increased risk of neurodevelopmental disorders in children

A new synthesis of global evidence finds that experiencing gestational diabetes during pregnancy is linked with a decline in intellectual function among mothers, and may increase the risk of neurodevelopmental disorders, including autism spectrum disorder and attention-deficit/hyperactivity disorder (ADHD) in children.

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Overweight and obesity don’t always increase the risk of an early death, Danish study finds

It is possible to be “fat but fit,” new research presented at the annual meeting of the European Association for the Study of Diabetes (EASD) held in Vienna, Austria (15–19 September) suggests.

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Maternal Dietary Diversity Lowers Childhood Eczema Risk: Study

A new study published in the journal of Pediatric Allergy and Immunology revealed that greater dietary diversity during pregnancy was associated with a reduced risk of atopic dermatitis in early childhood. The findings of the study emphasize on the potential protective role of maternal nutrition.

The research followed 677 mother-infant pairs from pregnancy through the children’s first 3 years, examined how maternal dietary diversity influenced the development of atopic dermatitis, while also considering the role of the infant gut microbiome.

To assess diet quality, this study used the Minimum Dietary Diversity for Women (MDD-W) guideline developed by the Food and Agriculture Organization. This measure evaluates whether a mother consumes foods from a broad range of groups, including whole grains, proteins, dairy, fruits, and vegetables, during pregnancy. Infant gut microbiota was analyzed at 2 months postpartum, and the incidence of atopic dermatitis in the children was monitored up to age three.

Also, while maternal diet did not appear to influence the overall diversity of the infant gut microbiome (alpha and beta diversity) it did alter its composition. The study highlighted 2 bacterial genera namely Rothia and Parabacteroides, both of which were linked to reduced risk of atopic dermatitis when present in greater abundance.

The analysis also revealed a connection between maternal nutrient intake and microbiome composition. Vitamin A-rich fruits and vegetables, like carrots, sweet potatoes, and leafy greens, were linked to higher levels of the Parabacteroides genus, which suggests a potential pathway through which maternal diet supports infant immune resilience.

The mothers with the lowest dietary diversity had the highest rates of children developing the condition, whereas the individuals with the broadest food group intake showed markedly lower incidence. Additional data demonstrated that while microbiome diversity metrics (alpha and beta diversity) were not significantly different across groups, shifts in microbial composition were consistently observed in association with maternal diet quality.

Overall, the findings suggest that maternal dietary diversity during pregnancy may help protect against atopic dermatitis in children, possibly through modulation of the infant gut microbiome. However, further studies are required to confirm these findings and clarify the biological mechanisms involved.

Source:

Huynh, L. B. P., Fan, H.-Y., Huang, S.-Y., Nam, N. N., Nguyen, T. T., & Chen, Y.-C. (2025). Influence of maternal dietary diversity during pregnancy on infant atopic dermatitis: Exploring the microbiome link in a Taiwanese birth cohort. Pediatric Allergy and Immunology: Official Publication of the European Society of Pediatric Allergy and Immunology, 36(9),. https://doi.org/10.1111/pai.70200

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Rheumatoid Arthritis Patients Face Higher Risk of Kidney Stones: Study

Kidney stone disease remains a growing health concern worldwide, with metabolic, inflammatory, and lifestyle factors all contributing to risk. New research now suggests that individuals with rheumatoid arthritis (RA) may be particularly vulnerable.

In a large population-based study involving nearly 20,000 participants, researchers found that patients with RA experienced more than double the incidence of kidney stones compared to those without the condition. The association persisted even after adjusting for common risk factors such as age, sex, and comorbidities, highlighting a potential link between systemic inflammation and stone formation. Rheumatoid arthritis is a chronic autoimmune disease characterized by persistent joint inflammation and systemic immune dysregulation. Researchers propose several possible mechanisms that could explain the elevated risk of nephrolithiasis in RA patients. Chronic inflammation may alter calcium and uric acid metabolism, leading to increased urinary excretion and crystal deposition. Additionally, medications frequently used in RA management—such as corticosteroids and diuretics—may contribute to stone formation by influencing fluid balance and renal handling of minerals. Reduced physical activity and dietary modifications in RA patients could also indirectly affect risk. Importantly, the study’s design limited causal inference. While the findings underscore a significant association, they do not establish that RA directly causes kidney stones. Instead, the results suggest that RA may act as a clinical marker of increased susceptibility, requiring further mechanistic and longitudinal research to clarify causality. Clinically, these findings carry important implications. Physicians caring for RA patients should be aware of the heightened risk of nephrolithiasis and consider proactive monitoring, especially in individuals with additional metabolic risk factors. Lifestyle strategies such as hydration, balanced diets, and metabolic screening may help mitigate risks. Overall, the study highlights the broader systemic burden of RA beyond joint disease, adding kidney stones to the list of potential comorbidities requiring integrated care.

Keywords
rheumatoid arthritis, kidney stones, nephrolithiasis, autoimmune disease, inflammation, metabolic risk, comorbidity, uric acid, corticosteroids, cohort study

Reference:

Yue, G., Yan, Y., Zeng, X. et al. Association between rheumatoid arthritis and kidney stones: A cross-sectional study of NHANES 2007–2020. Arthritis Res Ther 27, 141 (2025). https://doi.org/10.1186/s13075-025-03604-w

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Biparametric MRI Effective as Multiparametric MRI in Detecting Prostate Cancer: Study

A recent study published in the Journal of the American Medical Association found biparametric MRI to performs just as well as the current gold-standard multiparametric MRI in detecting clinically significant prostate cancer. The findings could reshape global diagnostic practices, particularly in health systems struggling with capacity and cost constraints.

Multiparametric MRI has long been considered the standard for diagnosing prostate cancer, combining T2-weighted, diffusion-weighted, and dynamic contrast–enhanced (DCE) sequences. While highly effective, this approach is resource-intensive and relies on gadolinium-based contrast, raising both logistical and financial barriers. Biparametric MRI excludes the DCE sequence and offers a faster and less costly alternative. 

From April 2022 to September 2023, this trial enrolled 555 biopsy-naive men with elevated prostate-specific antigen (PSA) levels or abnormal digital rectal exams. Conducted across 22 centers in 12 countries, the study analyzed data from 490 participants after exclusions. The men, with a median age of 65 years and median PSA of 5.6 ng/mL, underwent both biparametric and multiparametric MRI. The radiologists first reported on biparametric scans without access to DCE data, then assessed the full multiparametric images.

If either imaging modality suggested clinically significant cancer, patients received targeted biopsies, with or without additional systematic sampling. The trial was designed to test whether biparametric MRI was “noninferior” to multiparametric MRI, with a 5% margin set for acceptable difference.

The study found biparametric MRI help identify cancer in 29.2% of men (143 out of 490) while multiparametric MRI detected it in 29.6% (145 out of 490). The difference was a mere −0.4 percentage points, statistically insignificant and well within the noninferiority margin.

Detection rates of clinically insignificant cancers were also nearly identical. Biparametric MRI flagged 9.2% of men, when compared with 9.6% for multiparametric scans. Also, central quality control showed that 99% of all scans were diagnostically adequate, highlighting the reliability of the abbreviated technique.

With approximately 4 million prostate MRIs performed worldwide each year, the shift to biparametric imaging could free up scanner time, shorten waiting lists, and reduce costs without compromising care. Overall, the findings suggest that, provided imaging quality is maintained, biparametric MRI has the potential to become the new global standard in prostate cancer diagnosis. 

Reference:

Ng, A. B. C. D., Asif, A., Agarwal, R., Panebianco, V., Girometti, R., Ghai, S., Gómez-Gómez, E., Budäus, L., Barrett, T., Radtke, J. P., Kesch, C., De Cobelli, F., Pham, T., Taneja, S. S., Hu, J. C., Tewari, A., Rodríguez Cabello, M. Á., Dias, A. B., Mynderse, L. A., … PRIME Study Group Collaborators. (2025). Biparametric vs multiparametric MRI for prostate cancer diagnosis: The PRIME diagnostic clinical trial. JAMA: The Journal of the American Medical Association. https://doi.org/10.1001/jama.2025.13722

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Low Hb Levels may Increase Risk of Diabetic Retinopathy in Adults with Type 2 Diabetes: Study

A recent study published in the journal of Eye revealed that low hemoglobin levels may significantly raise the risk of developing diabetic retinopathy (DR). The findings highlight hemoglobin as an overlooked but potentially modifiable risk factor in managing type 2 diabetes.

The research used the TriNetX network from 2010 to 2022 and analysed data from over 57,000 adults aged 45 years and older with type 2 diabetes. The participants were divided into a low hemoglobin (LHB) group, defined as 8–12 g/dL, and a control group with levels ≥12 g/dL. To ensure balanced comparisons, propensity score matching was employed to account for confounding factors such as age, sex, and comorbidities.

The study tracked the onset of new DR cases within a 6 to 36 month follow-up period. The results showed a striking increase in risk among the individuals with lower hemoglobin. Specifically, the LHB group underwent a 33% higher risk of developing overall DR when compared with controls. When broken down into subtypes, the risk of proliferative DR was nearly doubled (HR: 1.95). Non-proliferative DR, a less severe but still concerning stage, also showed a modest yet significant risk increase of 19% (HR: 1.19).

The study found that even mild reductions in hemoglobin (10–12 g/dL) were enough to elevate DR risk, suggesting that attention should not be limited to only severe anemia cases. Subgroup analysis shed further light on this relationship. The association between low hemoglobin and DR was stronger in patients without hypertension (HR: 1.58 vs. 1.21 in hypertensive individuals).

It also remained consistent regardless of blood sugar control, as measured by HbA1c, highlighting hemoglobin as an independent factor. Also, men appeared more vulnerable than women, showing a more pronounced association.

Overall, these findings have important implications for clinical practice. While blood glucose and blood pressure management remain cornerstones of diabetes care, the study suggests that routine monitoring and optimisation of hemoglobin levels could play a critical role in preventing sight-threatening complications.

Source:

Hung, K.-C., Chang, L.-C., Chang, Y.-J., Hsu, C.-W., Yew, M., Wu, J.-Y., & Chen, I.-W. (2025). Association between haemoglobin levels and the risk of diabetic retinopathy in adults with type 2 diabetes: a retrospective cohort study using the TriNetX network. Eye, 1–8. https://doi.org/10.1038/s41433-025-03982-0

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