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Abstract Drinking water contaminated by pathogenic micro-organisms increases the risk of infectious gastrointestinal disease which could potentially lead to acute kidney injury and even death, particularly amongst the young and the elderly. Earlier studies have shown a substantial reduction in the incidence of diarrheal disease over a period of one year using a polysulfone membrane water gravity-powered water filtration device. The current report is a continuation of these studies to assess the long-term effects of the innovative method on diarrheal incidence rates over a 4-year follow-up period. This follow-up study monitored the trend of self-reported diarrheal events in all households in the previously studied villages for 5 months, in the last half of each study year, using the same questionnaire utilized in the earlier study. Three villages that had no device yet installed served as controls. We computed monthly diarrheal incidence rates for all study years (standardized to per 100 person-months) and compared these to the pre-device incidence rate in 2018 and in the control group, using the Wilcoxon rank sum exact test. The average diarrheal incidence rates of 1.5 p100pm in 2019, 2.19 p100pm in 2021, and 0.54p100pm in 2022 were significantly different from an earlier study that reported 17.8 p100pm rates before the devices were installed in 2018, (all p-values

Abstract The Laser Interferometer Space Antenna (LISA) will be a transformative experiment for gravitational wave astronomy, and, as such, it will offer unique opportunities to address many key astrophysical questions in a completely novel way. The synergy with ground-based and space-born instruments in the electromagnetic domain, by enabling multi-messenger observations, will add further to the discovery potential of LISA. The next decade is crucial to prepare the astrophysical community for LISA’s first observations. This review outlines the extensive landscape of astrophysical theory, numerical simulations, and astronomical observations that are instrumental for modeling and interpreting the upcoming LISA datastream. To this aim, the current knowledge in three main source classes for LISA is reviewed; ultra-compact stellar-mass binaries, massive black hole binaries, and extreme or interme-diate mass ratio inspirals. The relevant astrophysical processes and the established modeling techniques are summarized. Likewise, open issues and gaps in our understanding of these sources are highlighted, along with an indication of how LISA could help making progress in the different areas. New research avenues that LISA itself, or its joint exploitation with upcoming studies in the electromagnetic domain, will enable, are also illustrated. Improvements in modeling and analysis approaches, such as the combination of numerical simulations and modern data science techniques, are discussed. This review is intended to be a starting point for using LISA as a new discovery tool for understanding our Universe.

We carry out three-dimensional computations of the accretion rate onto an object (of size R _sink and mass m ) as it moves through a uniform medium at a subsonic speed v _∞ . The object is treated as a fully absorbing boundary (e.g., a black hole). In contrast to early conjectures, we show that for an accretor with ${R}_{\mathrm{sink}}\ll {R}_{A}=2{Gm}/{v}_{\infty }^{2}$ in a gaseous medium with adiabatic index γ = 5/3, the accretion rate is independent of Mach number and is determined only by m and the gas entropy. Our numerical simulations are conducted using two different numerical schemes via the Athena++ and Arepo hydrodynamics solvers, which reach nearly identical steady-state solutions. We find that pressure gradients generated by the isentropic compression of the flow near the accretor are sufficient to suspend much of the surrounding gas in a near-hydrostatic equilibrium, just as predicted from the spherical Bondi–Hoyle calculation. Indeed, the accretion rates for steady flow match the Bondi–Hoyle rate, and are indicative of isentropic flow for subsonic motion where no shocks occur. We also find that the accretion drag may be predicted using the Safronov number, Θ = R _A / R _sink , and is much less than the dynamical friction for sufficiently small accretors ( R _sink ≪ R _A ).

Introduction: More men than women start kidney replacement therapy (KRT) although the prevalence of chronic kidney disease (CKD) is higher in women than men. We therefore aimed at analyzing sex-specific differences in clinical outcomes among 8237 individuals with CKD in stages 3 to 5 from Brazil, France, Germany, and the United States participating in the Chronic Kidney Disease Outcomes and Practice Patterns Study (CKDopps). Methods: Fine and Gray models, evaluating the effect of sex on time to events, were adjusted for age, Black race (model A); plus diabetes, cardiovascular disease, albuminuria (model B); plus estimated glomerular filtration rate (eGFR) slope during the first 12 months after enrollment and first eGFR after enrollment (model C). Results: There were more men than women at baseline (58% vs. 42%), men were younger than women, and men had higher eGFR (28.9 ± 11.5 vs. 27.0 ± 10.8 ml/min per 1.73 m2). Over a median follow-up of 2.7 and 2.5 years for men and women, respectively, the crude dialysis initiation and pre-emptive transplantation rates were higher in men whereas that of pre-KRT death was more similar. The adjusted subdistribution hazard ratios (SHRs) between men versus women for dialysis were 1.51 (1.27–1.80) (model A), 1.32 (1.10–1.59) (model B), and 1.50 (1.25–1.80) (model C); for pre-KRT death, were 1.25 (1.02–1.54) (model A), 1.14 (0.92–1.40) (model B), and 1.15 (0.93–1.42) (model C); for transplantation, were 1.31 (0.73–2.36) (model A), 1.44 (0.76–2.74) (model B), and 1.53 (0.79–2.94) (model C). Conclusion: Men had a higher probability of commencing dialysis before death, unexplained by CKD progression alone. Although the causal mechanisms are uncertain, this finding helps interpret the preponderance of men in the dialysis population.

Abstract Background 5q spinal muscular atrophy (SMA) is a disabling and life-limiting neuromuscular disease. In recent years, novel therapies have shown to improve clinical outcomes. Yet, the absence of reliable biomarkers renders clinical assessment and prognosis of possibly already affected newborns with a positive newborn screening result for SMA imprecise and difficult. Therapeutic decisions and stratification of individualized therapies remain challenging, especially in symptomatic children. The aim of this proof-of-concept and feasibility study was to explore the value of 1H-nuclear magnetic resonance (NMR)-based metabolic profiling in identifying non-invasive diagnostic and prognostic urinary fingerprints in children and adolescents with SMA. Results Urine samples were collected from 29 treatment-naïve SMA patients (5 pre-symptomatic, 9 SMA 1, 8 SMA 2, 7 SMA 3), 18 patients with Duchenne muscular dystrophy (DMD) and 444 healthy controls. Using machine-learning algorithms, we propose a set of prediction models built on urinary fingerprints that showed potential diagnostic value in discriminating SMA patients from controls and DMD, as well as predictive properties in separating between SMA types, allowing predictions about phenotypic severity. Interestingly, preliminary results of the prediction models suggest additional value in determining biochemical onset of disease in pre-symptomatic infants with SMA identified by genetic newborn screening and furthermore as potential therapeutic monitoring tool. Conclusions This study provides preliminary evidence for the use of 1H-NMR-based urinary metabolic profiling as diagnostic and prognostic biomarker in spinal muscular atrophy.

BackgroundSystematic analyses about sex differences in wait-listing and kidney transplantation after dialysis initiation are scarce. We aimed at identifying sex-specific disparities along the path of kidney disease treatment, comparing two countries with distinctive health care systems, the US and Austria, over time.MethodsWe analyzed subjects who initiated dialysis from 1979–2018, in observational cohort studies from the US and Austria. We used Cox regression to model male-to-female cause-specific hazard ratios (csHRs, 95% confidence intervals) for transitions along the consecutive states dialysis initiation, wait-listing, kidney transplantation and death, adjusted for age and stratified by country and decade of dialysis initiation.ResultsAmong 3,053,206 US and 36,608 Austrian patients starting dialysis, men had higher chances to enter the wait-list, which however decreased over time [male-to-female csHRs for wait-listing, 1978–1987: US 1.94 (1.71, 2.20), AUT 1.61 (1.20, 2.17); 2008–2018: US 1.35 (1.32, 1.38), AUT 1.11 (0.94, 1.32)]. Once wait-listed, the advantage of the men became smaller, but persisted in the US [male-to-female csHR for transplantation after wait-listing, 2008–2018: 1.08 (1.05, 1.11)]. The greatest disparity between men and women occurred in older age groups in both countries [male-to-female csHR for wait-listing after dialysis, adjusted to 75% age quantile, 2008–2018: US 1.83 (1.74, 1.92), AUT 1.48 (1.02, 2.13)]. Male-to-female csHRs for death were close to one, but higher after transplantation than after dialysis.ConclusionsWe found evidence for sex disparities in both countries. Historically, men in the US and Austria had 90%, respectively, 60% higher chances of being wait-listed for kidney transplantation, although these gaps decreased over time. Efforts should be continued to render kidney transplantation equally accessible for both sexes, especially for older women.

Abstract. Background. Despite a growing population of patients starting hemodialysis in China, little is known about markers of mineral bone disease (MBD) and their management. We present data on prevalence and correlates of hypocalcemia, hyperphosphatemia, and secondary hyperparathyroidism from the China Dialysis Outcomes and Practice Patterns Study (DOPPS), with evaluation of whether these laboratory markers triggered changes in management. Methods. We compared the frequency of measurement and prevalence of poor control of MBD markers in China DOPPS with other DOPPS regions. We also used generalized estimating equations to assess correlates of MBD markers, and separate models to assess predictors of vitamin D and phosphate binder prescriptions in the China DOPPS. Results. Severe hyperphosphatemia (>7 mg/dL) and secondary hyperparathyroidism (>600 pg/mL) were common (27% and 21% prevalence, respectively); both were measured infrequently (14.9% and 3.2% of patients received monthly measurements in China). Frequency of dialysis sessions was positively associated with hyperphosphatemia; presence of residual kidney function was negatively associated with both hyperphosphatemia and secondary hyperparathyroidism. Laboratory measures indicating poor control of MBD were not associated with subsequent prescription of active vitamin D or phosphate binder. Conclusions. There are substantial opportunities for improvement and standardization of MBD management in China. Development of country-specific guidelines may yield realistic targets and standardization of medication use accounting for availability and cost.

Rationale & Objective: Normalization of parathyroid hormone (PTH), serum calcium, and phosphorus levels may prevent coronary and bone disease in hemodialysis (HD) patients. We describe the trajectory of these mineral bone disorder parameters and treatments during the first 5 years of HD by international region and race. Study Design: Prospective cohort study. Setting & Participants: 33,517 US black/African American, US non-black/African American, European, and Japanese HD patients from the Dialysis Outcomes and Practice Patterns Study (DOPPS) phases 4 to 5 (2009-2015). Predictor: Time since HD initiation. Outcomes: Monthly cross-sections of mineral bone disorder parameters (PTH, serum calcium, and phosphorus) and medications (cinacalcet, active vitamin D, and phosphate binders). Results: Mean PTH levels declined precipitously during the first 4 months of HD in all 4 groups, then steadily increased during the next 4.5 years in the United States/Europe but not in Japan. 3 years after HD initiation (month 36), mean PTH level was highest in US black/African Americans (496 pg/mL), despite greater prescription of cinacalcet (23%) and active vitamin D (85%), and lowest in Japan (151 pg/mL). Mean serum calcium and phosphorus levels increased during the first 4 months of HD. By month 36, the mean calcium level was lower in Japan (8.8 mg/dL) than United States/Europe (9.0-9.1 mg/dL), while the mean phosphorus level was lower in Europe (4.8 mg/dL) than United States/Japan (5.1-5.3 mg/dL). Limitations: Lack of data for medication dosages; most patients were not followed from HD onset. Conclusions: Large differences exist in the levels, trajectories, and therapies for PTH, calcium, and phosphorus by country and race in the first 5 years of HD. Higher PTH levels were observed in the United States, especially among black/African American patients, despite greater use of cinacalcet and active vitamin D than in Japan or Europe. Potential contributors to differences in PTH levels should be explored to study their impact on PTH management strategies and consequent bone and cardiovascular complications. Index Words: Hemodialysis, mineral bone disorders, DOPPS, international comparisons, mineral markers

Abstract Background The putative benefits of cinacalcet therapy for management of secondary hyperparathyroidism (SHPT) are thought to be most manifested when patients are taking it consistently and as prescribed. Real-world descriptions of cinacalcet prescription discontinuation and reinitiation in European hemodialysis patients are lacking. To address this knowledge gap, we used Dialysis Outcomes and Practice Patterns Study (DOPPS) data, based on dialysis facility medical records, from seven European countries to estimate rates and predictors of cinacalcet prescription discontinuation and reinitiation in hemodialysis patients and to describe the trajectories of CKD-MBD laboratory values after discontinuation. Methods Cox regression analyses were used to predict (1) cinacalcet discontinuation among 613 patients with ≥3 consecutive months without cinacalcet prescription immediately prior to a new cinacalcet prescription and (2) cinacalcet reinitiation among 415 patients with a newly discontinued cinacalcet prescription immediately after ≥3 consecutive months of prescribed use. Results Cinacalcet was discontinued in 21 and 35% of new users after 6 and 12 months, respectively. Cinacalcet was reinitiated in 38 and 49% of newly-discontinued users after 6 and 12 months, respectively. Predictors of discontinuation included lower parathyroid hormone (PTH) in the previous month (

BackgroundChronic kidney disease (CKD) is less prevalent among men than women, but more men than women initiate kidney replacement therapy. Differences in CKD awareness may contribute to this gender gap, which may further vary by race/ethnicity. We aimed to investigate trends in CKD awareness and the association between individual characteristics and CKD awareness among US men versus women.Methods and findingsWe conducted a serial, cross-sectional analysis of 10 cycles (1999-2018) from the National Health and Nutrition Examination Survey (NHANES). Adult participants with CKD stages G3-G5 (estimated glomerular filtration rate [eGFR] ConclusionsCKD awareness is lower among US women than men. The narrowing gap between the sexes in more recent years and the results on CKD awareness by serum creatinine indicate that health care professionals have previously been relying on serum creatinine to inform patients about their condition, but in more recent years have been using eGFR, which accounts for women's lower serum creatinine levels due to their lower muscle mass. Additional efforts should be made to increase CKD awareness among both sexes.

Introduction: In China, a quarter of patients are undergoing 2-times weekly hemodialysis. Using data from the China Dialysis Outcomes and Practice Patterns Study (DOPPS), we tested the hypothesis that whereas survival and hospitalizations would be similar in the presence of residual kidney function (RKF), patients without RKF would fare worse on 2-times weekly hemodialysis. Methods: In our cohort derived from 15 units randomly selected from each of 3 major cities (total N = 45), we generated a propensity score for the probability of dialysis frequency assignment, estimated a survival function by propensity score quintiles, and averaged stratum-specific survival functions to generate mean survival time. We used the proportional rates model to assess hospitalizations. We stratified all analyses by RKF, as reported by patients (urine output

The sliding wear of black-fiber palm wood was first investigated under dry conditions against smooth steel. Tests were carried out in two different configurations, i.e. pin on disc and ring on plate. In both cases, the transverse, in plane fiber orientation resulted in the highest values of friction coefficient and specific wear rate. The normal and parallel fiber orientations showed a better performance. In all cases, the coefficient of friction was rather high (average range between 0.4 to 0.7) whereas the specific wear rates varied between 10-6 to 10-5 (mm3/Nm). The latter are comparable to those of short fiber reinforced thermoplastics. A following impregnation in various oils reduced the wear rates, and the friction coefficients went down to values between 0.1 and 0.3. The different efficiency of the various oils was discussed.

Monitoring phenylalanine (Phe) concentrations is critical for the management of phenylketonuria (PKU). This can be done in dried blood spots (DBS) or in EDTA plasma derived from capillary or venous blood. Different techniques are used to measure Phe, the most common being flow-injection analysis tandem mass spectrometry (FIA-MS-MS) and ion exchange chromatography (IEC). Significant differences have been reported between Phe concentrations in various sample types measured by different techniques, the cause of which is not yet understood. We measured Phe concentrations in 240 venous blood samples from 199 patients with hyperphenylalaninemia in dried blood spots, EDTA plasma and erythrocytes by FIA-MS-MS and IEC. Phe concentrations were significantly lower in erythrocytes than in plasma leading to about 19% lower Phe DBS concentrations compared with plasma independent from the method used for quantification. As most therapy recommendations for PKU patients are based on plasma concentrations reliable conversion of DBS into plasma concentrations is necessary. Variances of Phe concentrations in plasma and DBS are not linear but increases with higher concentrations indicating heteroscedasticity. We therefore suggest the slope of the 75th percentile from quantile regression as a correction factor.

Abstract Background Anemia management protocols in hemodialysis (HD) units differ conspicuously regarding optimal intravenous (IV) iron dosing; consequently, patients receive markedly different cumulative exposures to IV iron and erythropoiesis-stimulating agents (ESAs). Complementary to IV iron safety studies, our goal was to gain insight into optimal IV iron dosing by estimating the effects of IV iron doses on Hgb, TSAT, ferritin, and ESA dose in common clinical practice. Methods 9,471 HD patients (11 countries, 2009-2011) in the DOPPS, a prospective cohort study, were analyzed. Associations of IV iron dose (3-month average, categorized as 0,

Cobalamin C (cblC) defect is an inherited autosomal recessive disorder that affects cobalamin metabolism. Patients are treated with hydroxycobalamin to ameliorate the clinical features of early-onset disease and prevent clinical symptoms in late-onset disease. Here we describe a patient in whom prenatal maternal treatment with 30 mg/week hydroxycobalamin and 5 mg/day folic acid from week 15 of pregnancy prevented disease manifestation in a girl who is now 11 years old with normal IQ and only mild ophthalmic findings. The affected older sister received postnatal treatment only and is severely intellectually disabled with severe ophthalmic symptoms. This case highlights the potential of early, high-dose intrauterine treatment in a fetus affected by the cblC defect.

The low-carbon economy of the future needs low consumption of fossil and high quality renewable based fuels. This requires high efficiencies, good part-load performance and fuel flexibility. A very promising concept to achieve that is the combination of solid oxide fuel cells (SOFC) with a gas turbine (GT) in a pressurized hybrid power plant. However, experimental data for such SOFC/GT systems are rare. Thus, the DLR built a test rig to analyse such a system with 30 kW electrical output. A 30 kW SOFC module is used under pressurized conditions with components that emulate the GT. Commercially available stacks and state of the art peripheral components are installed. These include e.g. a hot anode off-gas recirculation blower, a steam reformer and recuperator. The system was put into operation and is used to experimentally analyse its operational behaviour. This publication will give insights about the current status of the experimental work. It will outline the basic SOFC/GT process, the implementation within the installed SOFC system and the degrees of freedom in comparison to a coupled system. Experimental results are shown and the impact of main parameters is analysed.

BackgroundA comprehensive analysis of sex-specific differences in the characteristics, treatment, and outcomes of individuals with end-stage renal disease undergoing dialysis might reveal treatment inequalities and targets to improve sex-specific patient care. Here we describe hemodialysis prevalence and patient characteristics by sex, compare the adult male-to-female mortality rate with data from the general population, and evaluate sex interactions with mortality.Methods and findingsWe assessed the Human Mortality Database and 206,374 patients receiving hemodialysis from 12 countries (Australia, Belgium, Canada, France, Germany, Italy, Japan, New Zealand, Spain, Sweden, the UK, and the US) participating in the international, prospective Dialysis Outcomes and Practice Patterns Study (DOPPS) between June 1996 and March 2012. Among 35,964 sampled DOPPS patients with full data collection, we studied patient characteristics (descriptively) and mortality (via Cox regression) by sex. In all age groups, more men than women were on hemodialysis (59% versus 41% overall), with large differences observed between countries. The average estimated glomerular filtration rate at hemodialysis initiation was higher in men than women. The male-to-female mortality rate ratio in the general population varied from 1.5 to 2.6 for age groups ConclusionsWomen's survival advantage was markedly diminished in hemodialysis patients. The finding that fewer women than men were being treated with dialysis for end-stage renal disease merits detailed further study, as the large discrepancies in sex-specific hemodialysis prevalence by country and age group are likely explained by factors beyond biology. Modifiable variables, such as catheter use, showing significant sex interactions suggest interventional targeting. Please see later in the article for the Editors' Summary.

The pathophysiology underlying new-onset diabetes after transplantation (NODAT) is unresolved. We obtained demographics and laboratory data from all 1064 renal transplant recipients followed at our outpatient clinic in 2009/2010, randomly assigned 307 patients without previously diagnosed diabetes to a routine 2-hour oral glucose tolerance test (OGTT), and compared the metabolic results to a large, unrelated cross-sectional cohort of non-transplanted subjects. Among renal transplant recipients, 11% had a history of NODAT, and 12% had type 1 and type 2 diabetes. 42% of all OGTTs were abnormal (9% diabetic), predominantly in older patients who received tacrolimus. Compared to non-transplanted subjects, basal glucose was lower and HbA1c higher in renal transplant patients. Compared to non-transplanted subjects, insulin secretion was inferior, and insulin sensitivity improved at ≥6 months, as well as 3 months post-transplantation:(The Figure shows linear spline interpolation; all p for overall difference between non-Tx and Tx patients

It is increasingly difficult to satisfy growing demands for spectrum with the conventional policy of fixed spectrum allocation. To overcome this problem, flexible/dynamic spectrum sharing methods that can significantly improve spectrum utilization of the spectrum have gained increasing interest recently. This paper presents two dynamic spectrum sharing approaches, a centralized and a decentralized one. The centralized approach is based on hierarchical trading. Each level of hierarchy is composed of “markets†that are associated with a certain spatial area and trading occurrence frequency, whereas area size and trading occurrence frequency depend on the hierarchy level. The decentralized approach is based on game-theory. There, it is assumed that the operators are averse to unequal payoffs and act unselfishly, enabling a stable and sustainable community. Numerical results show that, in the observed scenario, both proposals outperform the reference case of fixed resource allocation significantly in terms of utilized bandwidth. Whereas, negotiation costs for spectrum brokerage appear in the centralized approach, nonnegligible amounts of spectrum are lost in the decentralized approach due to collisions. Thus, a hybrid of centralized and decentralized approach that exploits the benefits of both is also considered.

This study explores the potential of 1H-NMR spectroscopy-based metabolic profiling in various biofluids as a diagnostic and predictive modality to assess disease severity in individuals with 5q spinal muscular atrophy. A total of 213 biosamples (urine, plasma, and CSF) from 153 treatment-naïve patients with SMA across five German centers were analyzed using 1H-NMR spectroscopy. Prediction models were developed using machine learning algorithms which enabled the patients with SMA to be grouped according to disease severity. A quantitative enrichment analysis was employed to identify metabolic pathways associated with disease progression. The results demonstrate high sensitivity (84–91%) and specificity (91–94%) in distinguishing treatment-naïve patients with SMA from controls across all biofluids. The urinary and plasma profiles differentiated between early-onset (type I) and later-onset (type II/III) SMA with over 80% accuracy. Key metabolic differences involved alterations in energy and amino acid metabolism. This study suggests that 1H-NMR spectroscopy based metabolic profiling may be a promising, non-invasive tool to identify patients with SMA and for severity stratification, potentially complementing current diagnostic and prognostic strategies in SMA management. [ABSTRACT FROM AUTHOR]

Zirconium mononitride (ZrN), among other nitrides, is of interest as a catalyst in general and especially for the electrochemical nitrogen reduction reaction (NRR). Synthesis of nanoparticulate ZrN is challenging due to the formation of undesired other phases and the need for hazardous precursors or high temperatures. The urea glass method (UGM) was used in this work to synthesise nanoparticulate zirconium nitride. A focused parameter variation of the precursor composition and process conditions lead to an optimised synthesis, greatly improving material purity in regard to minor phase and residual carbon content. The nitride starts to form at a pyrolysis temperature of 900 °C, likely by irreversible decomposition of previously‐formed oxynitride, which forms from amorphous ZrO2 and urea during pyrolysis. An increased pyrolysis temperature improves product purity but slightly lowers nitride purity. Calcium admixture slows the urea decomposition, yielding a more phase‐pure product with less residual carbon at low amounts. A classical carbothermal nitridation (CN) was used in comparison to the UGM to synthesise ZrN. This was successful in synthesising zirconium carbonitride Zr(N, C) without minor phase, but significant carbon remains if added overstoichiometrically. Using less carbon and a longer dwelling time yielded ZrN without significant carbide (ZrC) incorporation or residual carbon, but with some minor ZrO2 phase, proving a promising synthesis route. A limited electrochemical investigation of a synthesised ZrN material was carried out to scope the general material behaviour and to qualitatively derive the potential catalytic activity for the NRR. Higher reductive currents in nitrogen‐saturated electrolyte qualitatively point to NRR activity, but further and more in‐depth examination is required. [ABSTRACT FROM AUTHOR]

Infants born to mothers with phenylketonuria (PKU) may develop congenital abnormalities because of elevated phenylalanine (Phe) levels in the mother during pregnancy. Maintenance of blood Phe levels between 120 and 360 μmol/L reduces risks of birth defects. Sapropterin dihydrochloride helps maintain blood Phe control, but there is limited evidence on its risk–benefit ratio when used during pregnancy. Data from the maternal sub‐registries—KAMPER (NCT01016392) and PKUDOS (NCT00778206; PKU‐MOMs sub‐registry)—were collected to assess the long‐term safety and efficacy of sapropterin in pregnant women in a real‐life setting. Pregnancy and infant outcomes, and the safety of sapropterin were assessed. Final data from 79 pregnancies in 57 women with PKU are reported. Sapropterin dose was fairly constant before and during pregnancy, with blood Phe levels maintained in the recommended target range during the majority (82%) of pregnancies. Most pregnancies were carried to term, and the majority of liveborn infants were reported as 'normal' at birth. Few adverse and serious adverse events were considered related to sapropterin, with these occurring in participants with high blood Phe levels. This report represents the largest population of pregnant women with PKU exposed to sapropterin. Results demonstrate that exposure to sapropterin during pregnancy was well‐tolerated and facilitated maintenance of blood Phe levels within the target range, resulting in normal delivery. This critical real‐world data may facilitate physicians and patients to make informed treatment decisions about using sapropterin in pregnant women with PKU and in women of childbearing age with PKU who are responsive to sapropterin. [ABSTRACT FROM AUTHOR]