- Discussion
- 10.1038/s41591-025-04115-6
A blueprint to accelerate rare pediatric gene therapy approvals.
- Mar 25, 2026
- Nature medicine
- Mohamed Abou-El-Enein + 3 more +3
Publications from 2021 to 2026
Showing 10 of 854 papers
A blueprint to accelerate rare pediatric gene therapy approvals.
Nationwide assessment of labor induction at full-term for low-risk pregnancy in the United States from 2018 to 2022.
Role of labor induction requiring cervical preparation for full-term, low-risk pregnant patients is under active investigation. The objective of this study was to describe nationwide temporal trends, characteristics, and outcomes associated with labor induction requiring cervical preparation at full-term gestation for low-risk pregnant patients in the United States. This cross-sectional study queried the Agency for Healthcare Research and Quality mechanism's Healthcare Cost and Utilization Project National Inpatient Sample. Study population included 5,003,348 hospital deliveries at 39-406/7weeks' gestation for singleton, cephalic low-risk pregnancy from 2018 to 2022, excluding historically known presumed pre-labor pregnancy risk factors. Exposure was labor induction status, defined as the performance of cervical preparation (ripening or mechanical dilation), with or without adjunctive intervention (artificial rupture of membranes or oxytocin administration). Main outcome measures were set as cesarean delivery as the primary outcome; induction failure, chorioamnionitis, umbilical cord prolapse, in-labor uterine rupture, postpartum hemorrhage, severe maternal morbidity and mortality as the secondary outcomes, assessed with multivariable generalized linear model. A total of 618,785 (12.4%) deliveries had labor induction requiring cervical preparation. The rate increased from 8.8 to 14.4% between QT1/2018-QT2/2020 (P-trend < .001), at which the rate started gradually decreasing when the COVID-19 pandemic occurred (14.4% at QT2/2020 to 13.4% at QT4/2022, P-trend = .04). The majority of cervical preparation was ripening alone (69.9%), followed by mechanical dilation alone (20.7%) and both ripening and mechanical dilation (9.4%). During the 5-year study period, cervical preparation with mechanical dilation alone (14.4% to 26.9%) and both ripening and mechanical dilation (4.6% to 11.4%) increased (both, P-trend < .001); adjunctive use of both oxytocin and artificial rupture of membranes (18.7% to 27.0%) and oxytocin alone (15.0% to 18.8%) increased while artificial rupture of membranes alone decreased (29.5% to 23.0%) (all, P-trend < .001). When compared to 4,384,563 deliveries without labor induction cervical preparation, induction was associated with cesarean delivery (17.1% vs 9.2%, adjusted-incidence rate ratio [aIR] 1.75, 95% confidence interval [CI] 1.74-1.76), chorioamnionitis (3.2% vs 1.9%, aIR 1.67, 95%CI 1.64-1.69), umbilical cord prolapse (0.2% vs 0.1%, aIR 1.67, 95%CI 1.58-1.77), in-labor uterine rupture (21.8 vs 7.8 per 100,000, aIR 2.66, 95%CI 2.17-3.25), postpartum hemorrhage (5.7% vs 3.9%, aIR 1.42, 95%CI 1.40-1.44), severe maternal morbidity (0.4% vs 0.2%, aIR 1.61, 95%CI 1.54-1.68), and maternal mortality (4.8 vs 0.7 per 100,000, aIR 7.34, 95%CI 4.37-12.33). In conclusion, this nationwide cross-sectional assessment suggests the increasing trend of full-term labor induction for study-defined low-risk pregnancy may have stopped during the COVID-19 pandemic in the United States. The observed association with cesarean delivery and adverse outcomes related to labor induction requiring cervical preparation warrant further investigation.
Read moreSensory Over-Responsivity in Autism: A Bidirectional Brain-Gut-Microbiome Model.
The brain-gut-microbiome (BGM) system encompasses a bidirectional communication network between the gut microbiome and the brain and plays a critical role in maintaining gut function and modulating behavior. Here, we specifically focus on the interactions between sensory over-responsivity (SOR), a common phenotype in Autism Spectrum Disorder (ASD), and the BGM system and discuss potential mechanisms underlying these interactions. We examine key constructs and frameworks related to sensory processing, autism, and the BGM system, synthesize literature to identify knowledge gaps, and propose a model linking the BGM system and SOR in autism. Grounded in prior literature, we propose a conceptual model involving the brain, the gut microbiome, and SOR in autism. In this model sensory features of foods commonly associated with SOR contribute to atypical eating behaviors, resulting in differences in dietary intake, which in turn affect the gut and oral microbiomes. The gut microbiome can impact neural activity and brain structure via the connection between the gut and the brain, further contributing to SOR. The interdisciplinary nature of this topic is emphasized, bridging research from neuroscience, microbiology, pediatrics, nutrition, psychology, and occupational therapy/occupational science. Reflecting on the intersection of autism characteristics and microbiome health, this analysis offers critical insights for research and clinical practice.
Read more786 Homologous Recombination Deficient Prostate Cancer: A Molecular and Pathologic Study
Carc/Rarc Mapping Using AI Tools
CARC and RARC are two of the core code sets used in healthcare claims adjudication. They explain why a claim was paid, denied, or adjusted. Your search results confirm this across multiple authoritative sources. CARCs explain the reason for a financial adjustment on a claim or service line. They tell you why the payer changed the billed amount (denied, reduced, bundled, not covered, etc.). They appear on ERAs (Electronic Remittance Advice) and EOBs. They are standardized across the industry and maintained by X12. Remittance Advice Remark Codes (RARCs) are used to provide additional explanation for an adjustment already described by a Claim Adjustment Reason Code (CARC) or to convey information about remittance processing. Each RARC identifies a specific message as shown in the Remittance Advice Remark Code List. There are two types of RARCs, supplemental and informational. The majority of the RARCs are supplemental; these are generally referred to as RARCs without further distinction. Supplemental RARCs provide additional explanation for an adjustment already described by a CARC. The second type of RARC is informational.
Read moreDifferential Operators on the Base Affine Space of SLn and Quantized Coulomb Branches
Abstract We show that the algebra $D_\hbar (SL_{n}/U)$ of differential operators on the base affine space of $SL_{n}$ is the quantized Coulomb branch of a certain 3d $\mathcal{N} = 4$ quiver gauge theory. In the semiclassical limit this proves a conjecture of Dancer–Hanany–Kirwan about the universal hyperkähler implosion of $SL_{n}$. We also formulate and prove a generalization identifying the Hamiltonian reduction $T^{*} SL_{n}\ /\!/ _{\psi } U$ as a Coulomb branch for an arbitrary unipotent character $\psi $. As an application of our results, we provide a new interpretation of the Gelfand–Graev symmetric group action on $D_\hbar (SL_{n}/U)$.
Read moreGateway to Global Aging Data: Enabling Comparative Research on Aging, Health, and Environment
BackgroundThe Gateway to Global Aging Data (Gateway) is an open research platform supporting the Health and Retirement Study International Network of Studies (HRS‐INS), a global network comprising longitudinal and multidisciplinary surveys of older populations across 46 countries. The Gateway integrates survey metadata, harmonized data files, and advanced tools to facilitate comparative studies on aging, brain health, and risk factors. Three initiatives—the Data Enclave, the Policy Explorer, and the Environmental Exposome —further enhance the platform's capacity to study complex research questions.MethodThe Gateway delivers harmonized datasets and comprehensive survey metadata to enable cross‐national and longitudinal analyses of critical domains such as health, economic status, and social networks. The Data Enclave provides a secure, remote‐access computing environment for analyzing survey data while maintaining robust privacy protections and compliance with data‐sharing policies. In parallel, the Policy Explorer links individual‐level survey data to historical and current policy variables, empowering researchers to explore how macro‐level policy environments influence aging outcomes, and the Environmental Exposome integrates data on physical, natural, and chemical environments.ResultThe Gateway have facilitated significant advancements in aging research. Harmonized data have revealed cross‐national differences in aging experiences. The Data Enclave has enabled secure analyses of survey data, including cognitive health trends, while the Policy Explorer has supported research into the effects of retirement policies and social support systems on individual outcomes. The Environmental Exposome stimulate research on the effects of environmental factors on health and wellbeing.ConclusionThe Gateway to Global Aging Data serves as a critical resource for advancing cross‐national research on aging. Through the integration of harmonized data and innovative tools, the Gateway empowers researchers to uncover policy‐relevant insights, addressing global aging challenges and informing strategies to promote healthy and equitable aging worldwide.
Read more6 Organizational interventions to address burnout, moral injury, and grief in the homeless workforce
Cognitive functioning predicts vulnerability to financial exploitation in APOE e4 carriers.
An emerging body of work suggests that financial exploitation vulnerability (FEV) may be an early marker of Alzheimer's disease (AD). We examined how APOE e4 genotype and cognition at baseline interact to predict subsequent FEV one year later. Participants were 95 older adults without dementia aged 50 or older (M age = 69.09, SD = 7.51; 72.63 % female; 72.63 % White non-Hispanic). Participants completed two annual assessments that included comprehensive neuropsychological testing and a measure of FEV. Saliva samples for APOE genotyping were collected. Linear regression models regressed Year 2 FEV on APOE, global cognition, and their interaction. Age, sex, education, and Year 1 FEV scores were covaried. Models were re-run considering cognitive domains separately (memory, language, and attention/working memory and executive functioning). Main effects of APOE and cognition were not found. However, cognition and APOE status interacted to predict Year 2 FEV. Specifically, worse cognitive functioning predicted higher FEV only for e4 carriers. Cognitive domain analyses revealed more nuanced relationships. Findings suggest that cognition is a relevant risk factor of FEV for e4 carriers, and support the emerging idea that FEV may be an early marker of AD-related neuropathological changes.
Read moreP170. Radiographic and patient reported outcomes of lumbar interbody fusion using hydroxyapatite-infused polyetheretherketone cages: no significant advantage in adding BMP