The methods used to inform item generation in this study reflect best practice guidelines in the initial stages of questionnaire development [9], [10] and [11]. Gaining a rich and detailed understanding of the construct to be measured is best achieved from focused interviews with the relevant

population. Whilst this is particularly relevant for condition specific measures however, this generic measure needed to be applicable to people over a range of health conditions and roles (i.e. patients and carers). The opportunity to carry out http://www.selleckchem.com/products/dabrafenib-gsk2118436.html secondary data analysis using a large interview archive which spanned a range of conditions was therefore particularly useful for the development of this item pool. However, analysis of secondary data can be restrictive in comparison to primary research where the interviewer can focus their questions on the issues of most interest to their

own research agenda [15]. In some interviews the original reseracher had not probed into participants experiences of using health websites. Integrating secondary analysis of several, purposively selected collection of interviews with a conceptual literature review and using confirmatory sources of data was therefore vital in ensuring all Selleck Panobinostat potential themes were investigated thoroughly and assisted the triangulation of the findings. Secondary data analysis has also been critiqued for lacking relevant contextual knowledge when the researcher was not involved in the primary research. However, the availability of Y-27632 2HCl video and audio files of interviews largely overcomes this problem. Suitability of the data was also assessed through a number of steps before formal analysis commenced: (1) thematic summaries

and participant biographies prepared by the primary researchers were read, (2) primary researchers were consulted to gauge the appropriateness of the data for the research purpose, and (3) primary researchers coding books of relevant themes from their initial analyses were made available to the research team. Cognitive interviews also confirmed the relevance of the qualitative findings. Current studies evaluating ehealth interventions are limited by the lack of a suitable instrument to measure health-related effects associated with using a health website. A person may use guidance, filtering and accreditation tools [29] to help them assess health information on the internet. However, these instruments do not capture how a person may be affected through engaging with a website and users may be concerned of coming across factually correct, yet unwelcome information [30]. Furthermore, such accreditation tools fail to take into account that websites provide more than information, but can also be mechanisms of support. The potential effects of using health-related websites and support groups have been explored [31] using self-report measures which were not specifically developed to capture the range of effects associated with internet use.

, 2010). On the 40–160-h time scale the correlation this website relationship is cleaner for the model than observations, as model SST generally has less variability than observations ( Figs. 1b and 2). To examine the statistics

of that relationship, the lagged correlation is calculated for the filtered time series of both model and observations. Each value of the lagged correlation series is a calculation of correlation with the time series of SST and τ offset from one another by a different lead/lag time. We consider only the correlations in which the τ time series leads SST, because the ocean model is forced by a prescribed atmosphere that has no response to the ocean, rendering lag time meaningless. For comparison between model and observations, we select the largest magnitude correlation for any lead time less than 48 h. The lead time itself is examined separately. The KPP turbulent mixing scheme is implemented in a version of the Massachusetts Institute of Technology general circulation model (MITgcm) (Adcroft, 1995, Marshall et al., 1997a and Marshall et al., 1997b), in hydrostatic configuration

with a 1/3° resolution C-grid on a domain encompassing the Tropical Pacific, from 26°S to 30°N and 104°W to 290°W (Table 1). The model is run for approximately four years, from Nov 1st, 2003 to October 13th, 2007 with a 15-min timestep. The model configuration is based on Hoteit et al., 2008 and Hoteit et al., 2010. Initial and lateral boundary conditions for the ocean temperature, salinity, and velocity come from the OCean Comprehensible Atlas (OCCA) (Forget, 2010). Surface forcing GSK2656157 for temperature, specific humidity, shortwave and longwave radiation, wind (unless otherwise noted), and precipitation are interpolated to the model grid size and time step from the NCEP/NCAR 1.8°, six-hourly Reanalysis (Kalnay et al., 1996) and prescribed at the ocean surface. The MITgcm calculates

heat fluxes between the ocean and atmosphere. The default experiment (Exp. 0 [Table 2]) uses the NCEP/NCAR forcing and default KPP parameter values. An ensemble of 42 additional experiments is conducted (Table 2). In the first three experiments the KPP parameters are held at their default values while wind forcing is replaced with alternatives: ECMWF (Gibson et al., 1997), NOAA/CIRES Twentieth Edoxaban Century reanalysis (Compo et al., 2011), and NASA Cross-Calibrated Multi-Platform Ocean Surface Wind Velocity (Atlas et al., 1996) (Exp. 1–3 [Table 2]). In the next 19 experiments, KPP parameters are perturbed to artificially large and small values (Exp. 4–22 [Table 2]). An additional 20 experiments are conducted using wind forcing that is blended from the NCEP/NCAR, ECMWF, and NASA products (Exp. 23–42 [Table 2]). The blending is done using a mixture model to weight the contribution from each of the three wind products, resulting in a Dirichlet distribution of weighting with the highest probability being an equal weight for each product.

In contrast, all mice treated with Pa-MAP in both concentrations survived at the end of experiment. The same pattern

was observed in mice treated with ampicillin ( Fig. 1B). Mice weights were further evaluated in the beginning and at the end of experiment. Infected and untreated mice lost 5.5% of their body weight after 72 h of experiment. In contrast, mice treated with Pa-MAP at 1 mg kg−1 gained 0.8% of their body weight, similar to Pa-MAP at 5 mg kg−1, which gained 0.5% of their body weight during the same period. Non-infected mice gained slightly more body weight (2.7%) compared to the Pa-MAP treatment groups. Infected mice treated with ampicillin at 2 mg kg−1 also had lost weight, equivalent to 5.6% of their initial body weight ( Fig. 1C and D). Some cytokines were evaluated selleckchem in attempt click here to identify an immunomodulatory effect of Pa-MAP in the mice immunologic system. This evaluation of immunomodulatory activity in vivo was investigated by quantification of IL-10, IL-12, TNF-α and NO in serum. Pa-MAP used as treatment

was evaluated at 1 mg kg−1, corresponding to a concentration of twice the minimum inhibitory concentration (MIC) of 512 μg mL−1 [34], and 5 mg kg−1, corresponding 10 times the MIC encountered in early study with Pa-MAP. Ampicillin at 2 mg kg−1 was used as a positive control. These concentrations of Pa-MAP were unable to modify IL-10 release when compared to the non-infected and untreated mice group. Similar data were observed for IL-12 and TNF-α production in all treatments groups

(Supplementary Fig. 1). Figure options Download full-size image Download as PowerPoint slide Antimicrobial resistance mechanisms developed by bacteria is a serious worldwide threat to public health, particularly for immunocompromised patients and those under immunosuppression therapy, e.g. patients oxyclozanide after organ transplant [29]. Moreover, infections caused by antibiotic-resistant microorganisms have contributed to increases in patient mortality, especially for those whose treatment with currently available drugs has become less efficient [14]. Due to these facts, peptides with antimicrobial activities have become extremely attractive for microorganisms control, mainly due their low toxicity effects into mammalian cells [24]. In our study, an alanine-rich peptide designed from a polar fish, P. americanus, with two repeat antifreeze motifs and clear in vitro deleterious activity against E. coli, with identical purification degree (see Fig. 1A) previously reported by Migliolo et al. [34] was evaluated in vivo. Some peptides were designed to develop a multifunctional product, able to eliminate microbes and increase the immune response, involving systematic variations in the structure of a base molecule, i.e. cationic charge, hydrophobicity and hydrophobic moment [21]. Moreover, some cationic peptides are known to be able to induce some immunomodulatory effect [37] and [62].

The red striped mullet is an extremely rare fish species in the Baltic Sea. The specimen collected in the Pomeranian Bay was identified

as Mullus surmuletus, although some characters were typical of M. barbatus. Nonetheless, the specimen’s identity was confirmed by Franz Uiblein (personal communication) as a ‘North-Sea’ form of M. surmuletus. There is a considerable lack of basic systematic and taxonomic knowledge on goatfishes, intraspecific morphological variation and genetic differentiation, and further detailed studies are required ( Uiblein 2007). There is considerable variation in the Mullus genus, even among populations from neighbouring habitats, which to some extent may reflect phenotypic plasticity ( Uiblein et al. 1998). Much more information may still be hidden behind morphological differentiation, if a specimen of Mullus from the MLN8237 price Skagerrak exhibiting a head shape intermediate between red mullet M. barbatus and striped red mullet M. surmuletus is anything to go by. Fage (1909, after Uiblein 2007) distinguished southern and northern forms of striped red mullet based mainly on head shape. There have also been problems with the correct identification of learn more Mullus spp. during regular bottom trawls in the North Sea. Additional confusion may arise from the continued usage of the common name ‘red mullet’ for both species. Recently, a detailed comparison of Mullus

specimens from the North Sea was started as part of an intended revision of the genus ( Uiblein 2007). Mullus either surmuletus has the status of RA (rare) on the HELCOM (2007) List of Species not threatened in the Baltic, its region of distribution

being in the Skagerrak, Kattegat and western Baltic. M. surmuletus is on the list of fish species occurring in German North Sea and western Baltic waters ( Ehrich et al. 2006); the frequency of occurrence in the total number of hauls in the former region is 6.05%; in the latter one it is low (0.98%). Lampart-Kałużniacka et al. (2007) reported the occurrence of 3 individuals of Mullus, identified as M. barbatus in Polish coastal waters (between 1998 and 2000, between the Kołobrzeg and łeba fishing grounds). Grygiel (2009) reported the presence of one specimen of striped red mullet in catches from open Baltic waters (56°N, 17°30′E) in 2007, and Skóra (2007) also reported one specimen from the Gulf of Gdańsk. Temperature increases and longer warming up periods may induce M. surmuletus to migrate to higher latitudes in the North Sea. Isolated occurrences of this species in the Norwegian Sea at 60°N have been documented ( Uiblein 2007). In the North Sea it was not caught by international bottom trawl surveys before 1988, but an ongoing northward shift in its distribution has been demonstrated since, with steadily increasing abundance in south-western areas ( Beare et al. 2004). This change in distribution and abundance has happened during a phase when temperature rises have taken place as a result of global climate change ( Hulme et al. 2002).

1 ratio of pLNT–SFFV–WPRE–cDNA, the packaging plasmid psPAX2 and pMD2.G using calcium phosphate transfection. After 18 h medium was replaced with 6 ml fresh medium. Twenty four hours later, virus was harvested and new medium added for a final period of 24 h. Virus containing supernatants were stored at 4 °C, pooled and filtered through 0.2 μm. Virus stocks were concentrated 100 fold by centrifugation for 2 h at 25,000 rpm in a SW-28 rotor. The concentration procedure removed HEK293T medium which was detrimental to normal RBL-2H3 growth. Virus was resuspended in 0.01 volume RBL-2H3 medium supplemented with 20 mM Hepes pH 7.6, aliquotted Luminespib datasheet and stored at −80 °C. Virus efficiency

was determined by titration on HEK293T and RBL-2H3 cells through fluorescence microscopy and western blot detection. RBL-2H3 cells stably expressing lentiviral constructs were made by infecting a 50% confluent 10 cm dish with

10 μl of concentrated virus in the presence of 6 μg ml−1 polybrene in 10 ml medium. Medium was replaced after 24 h and cells were allowed to expand for another 24 h. Cells were then cultured and evaluated for expression. Typical transduction efficiencies were 70%. Cells were collected after two weeks for FACS sorting and we sorted a 99% positive cell population of at least 5 × 105 cells using a FACSAria (BD). Cells were then transferred learn more to 10 cm dishes for maintenance culture. Lentiviruses reverse transcribe their RNA and integrate the newly made DNA into the chromatin (Bukrinsky

et al., 1993). In our hands selection or resorting was not needed within 2 months after transduction, since expression levels remained constant during this period. If expression drops however, cells can easily be sorted as above to enrich for transfectants with higher expression. For Western blot, 1 × 106 RBL-2H3 cells were lysed in 250 μl 1% Triton X-100, 100 mM NaCl, 50 mM Tris–HCl pH 7.6. Lysates were cleared by centrifugation and collected in 5× Laemmli buffer containing 10% SDS, 50% glycerol, 0.625 M Tris–HCl pH 6.8, 250 mM DTT 0.01% Bromophenol blue. Samples were electrophoresed Thalidomide through 7.5% SDS-PAA gels and blotted on Immobilon-FL PVDF (Millipore). Blots were analyzed for munc13-4, YFP and actin using primary antibodies described above. Secondary alexa680 or IrDye800 fluorescent antibodies were used for detection in the Odyssey imaging system (Li-Cor). Antibody incubations were typically 45 min, followed by three washing steps in blocking buffer (Li-Cor). siRNA depletion of endogenous rat munc13-4 in RBL-2H3 cells was done through a sequence targeting the ORF of rat munc13-4; GGAACAAGAUUUUUCACAAtt (Applied Biosystems). The siRNA was introduced using AMAXA nucleofection (Lonza), 100 μl buffer T, protocol X-001 according to manufacturers’ instructions. 1 × 106 cells were transfected with 20 μl 20 μM oligo (400 pmol), seeded in 2 ml full medium in a 6 cm dish and grown for 48 h, knock-down efficiency was typically over 90%.

, 2010). Van Maele-Fabry et al., 2006, Van Maele-Fabry

et al., 2007 and Van Maele-Fabry et al., 2008 pointed out exposure to pesticides as a possible risk factor for prostate cancer and leukemia by a meta-analysis of risk estimates in pesticide manufacturing workers. In a series of agricultural health studies, Lee et al., 2004a, Lee et al., 2004b and Lee et al., 2007 found an association between exposure to pesticides and cancer incidence, particularly lymphohematopoietic cancers for alachlor, lung cancer for Protein Tyrosine Kinase inhibitor chlorpyrifos, and colorectal cancer for aldicarb. Nowadays, chronic low-dose exposure to pesticides is considered as one of the important risk factors for cancer expansion. Therefore, carcinogenicity tests are now applied to detect carcinogenic potential of pesticides before allowing them to be marketed. Carcinogenicity testing is a long-term (around two years) rodent bioassay using two species of both sexes. According to a new list of Chemicals Evaluated for Carcinogenic Potential by EPA’s Pesticide Program published in 2010, more than

70 pesticides have been classified as a probable or possible carcinogen. This classification has been accomplished based on the information extracted from animal studies, metabolism studies, Rigosertib order structural

Avelestat (AZD9668) relationship with other carcinogens, and if available, epidemiologic findings in human (http://www.epa.gov/pesticides/carlist/). Carcinogenic properties of pesticides can be influenced by a series of complex factors including age, sex, individual susceptibility, amount and duration of exposure, and simultaneous contacts with other cancer causing chemicals. However, carcinogenic mechanisms of pesticides can be explored in their potential to affect genetic material directly via induction of structural or functional damage to chromosomes, DNA, and Histone proteins, or indirectly disrupting the profile of gene expression through impairment of cellular organelles like mitochondria and endoplasmic reticulum, nuclear receptors, endocrine network, and the other factors involved in maintenance of cell homeostasis (George and Shukla, 2011 and Rakitsky et al., 2000). Table 1 is indicating data extracted from epidemiological studies implicating on the relation between exposure to specific pesticides and increased risk of some kind of cancers. Birth defects or congenital disorders are defined as structural or functional abnormalities existing at birth or before birth that causes physical or mental disabilities.

There have been a number of attempts to redesign these enzymes to use the non-phosphorylated

donor, dihydroxyacetone (DHA), by using directed evolution [25] or rational methods using point mutations to redesign the phosphate binding pocket [26•]. In this respect fructose-6-phosphate aldolase (FSA) is of great interest as it has been shown to utilize multiple donor substrates such as dihydroxyacetone (DHA), hydroxyacetone and hydroxybutanone [27]. FSA also provides a route to the production of iminocyclitols which are attractive drug candidates [28]. FSA has been the subject of many studies to alter its substrate specificity Cisplatin manufacturer for different acceptor aldehydes and to increase its affinity for the specific donor DHA [29• and 30]. Another enzyme that uses DHA rather than DHAP is transaldolase (Tal) and, interestingly, FSA activity has been conferred on this enzyme by replacement of a single phenylalanine by tyrosine (F178Y) in the active site [31]. This F178Y variant has also been the subject of further study to increase its activity

with non-phosphorylated acceptor aldehydes. Structure-guided mutagenesis identified residues in the phosphate binding pocket that, when mutated, prevent phosphorylated acceptors from binding. This has produced an enzyme that can synthesize polyhydroxylated, non-phosphorylated compounds and be used in enzymatic cascade synthesis of this type of compound [32]. Many enzymes have Sorafenib solubility dmso been shown to have catalytic promiscuity and as well as Thymidylate synthase using engineering to subvert the substrate specificity of natural aldolases, attempts are now being made to enhance the catalytic promiscuity of other enzyme classes to produce novel aldolases. An early example of the conversion of one enzyme activity into another

type of reaction was the conversion of an alanine racemase into an aldolase by a single active site point mutation [33]. This variant enzyme catalysed a reaction similar to threonine aldolase with rates and specificities comparable with the native enzyme. More recently 4-oxalocrotonate tautomerase (4-OT) was shown to be promiscuous in having low aldolase activity towards the condensation of acetaldehyde and benzaldehyde to yield cinnamaldehyde. This low activity has been enhanced by a single point mutation, F50A, which increased the kcat/KM for the aldolase activity by 600-fold compared to that of the wild-type [ 34•]. Lipases have also been reported to display promiscuous aldolase activity [35 and 36] and recently asymmetric aldol reactions between acetone and 4-nitrobenzaldehyde (catalysed by porcine pancreas lipase) [37] and aromatic and heteroaromatic aldehydes with cyclic ketones (catalysed by chymopapain, nuclease p1, alkaline protease BLAP and acidic protease AUAP) [38 and 39] have been described.

5%. The most prevalent specific causative agent noted in the ISRIB molecular weight cultures was Staphylococcus aureus (25.8%). The least prevalent agents among the reported causative microorganisms were Enterobacter cloacae and Acinetobacter anitratus (0.9% each) ( Table 2). Of the total confirmed positive cultures, 13% had not been tested for resistance to antibiotics. Of the remaining 385 cultures, 87% were resistant to at least one type of antibiotic. The most commonly

reported resistance was to ampicillin (19.2%), followed by ticarcillin/clavulanic acid resistance (15.1%). Of the 445 total infected patients, 125 (28.1%) were matched with a comparison group of uninfected patients. Although the matching was perfect for gender, in 9 of 125 pairs, the patients fell into adjacent age groups. For these 9 pairs, the differences in actual age ranged between 2 and 12 years (M = 7.89, SD = 3.69). Of the 125 pairs, 56.8% were male (n = 142); of the total matched pairs, 45% were over

60 years of age, and of 125 pairs, 63.2% (n = 46) were matched based on the same admission unit. Nearly 42% of the infected patients had at least one cardiovascular disorder (e.g., hypertension, heart failure, or coronary artery disease) compared to 32% of the uninfected group. More than one-fourth of the infected patients had undergone at least one type of invasive procedures (e.g., endoscopy, bronchoscopy, nasogastric intubation, endotracheal intubation, colonoscopy, endoscopy, abdominal paracentesis, and/or bone marrow biopsy). The

leading comorbidity that was associated with an increased risk of Selleck Neratinib HCABSIs was “renal failure” (RR = 2.9, 95% CI: 1.6, 5.4). Blood products recipients experienced the greatest risk for HCABSIs (RR = 17.9, 95% CI: 4.2, 77.2) ( Table 3). Using a conditional logistic Ixazomib mw regression analysis, three models were examined (Table 4). Model 1 represented the Odds Ratios (OR), which are adjusted for matching factors but not for other risk factors. The four retained factors were “Blood Products”, “Invasive Procedures”, “Renal Failure”, and “Other Infections”. Model 2 adjusted for matching factors and all other factors in the full model. In Model 3, we dropped “other infections” as the four-factor, which was associated with a wide 95% confidence interval (1.9–243.9). The pseudo R2 for Models 2 and 3 were 0.40 and 0.33, respectively. This study is the first to provide insight into the epidemiology of HCABSIs in a large Jordanian hospital. The study estimated that the overall incidence for HCABSIs among adult hospitalized patients was 8.1 infections per 1000 admissions. This rate was similar to the incidence (8.5 infections per 1000 admissions) reported in Saudi Arabia [33] and much lower than the rate reported in an Egyptian study (76 per 1000 ICU admissions) [34].

According to these PK analysis, TDM results on day 2 can be evaluable as a steady state in patients with a normal renal function

and mild renal dysfunction. Tanigawara et al. reported that ABK clearance was related to Ccr, age, and body weight. LEE011 concentration The volume of distribution was different in healthy subjects and infected patients, and this difference was more pronounced among disease types [13]. Ikeda et al. [14] reported that duration time of infusion, Ccr, body mass index (BMI), serum albumin level, and presence of chronic heart failure were significant factors influencing Cpeak. Based on these findings, frequent follow-up TDM is recommended for patients with severe infection, impaired renal function, obesity or underweight, concomitant use of nephrotoxic agents (aminoglycosides, amphotericin B, cyclosporine, contrast media, etc.), and particular clinical conditions which cause fluctuating volumes of distribution. In a nationwide questionnaire survey (203 institutions) concerning TDM of ABK, Cmax was used in 88 institutions, and Cmin was used in 79 institutions as the target serum

concentrations that indicate clinical efficacy [15]. Although previous reports mainly analyzed based on Cmax, recent studies used Cpeak as an indicator of clinical efficacy [4], [9], [10], [11], [12], [16] and [17]. Regarding the optimum administration method of ABK based on the PK-PD theory, it has been reported that the trough concentration (OR = 2.00) and patient’s age (OR = 1.06) were indices of the development selleck chemical of renal dysfunction on multiple logistic regression analysis. The mean

trough concentrations were 2.6 μg/mL in patients with developing nephrotoxicity and 0.5 μg/mL in patients without nephropathy [9]. Sato et al. described that incidences of nephrotoxicity were 2.5%, 5.2%, and 13.1% in patients with a trough value of 3-mercaptopyruvate sulfurtransferase 1 μg/mL, 2 μg/mL, and 5 μg/mL, respectively [4]. As for ototoxicity, Suzuki et al. demonstrated that there was no significant correlation between auditory brainstem response abnormality with either peak ABK concentration 20 μg/mL, trough concentration 4 μg/mL, or total dose100 mg/kg [18]. a. Clinical effect can be expected when the Cmax/MIC ratio was 8 or higher, and target Cpeak of 15–20 μg/mL is recommend (C1-III). In studies using Cmax as an indicator of clinical efficacy in patients with once daily administration at the approved dose of 150–200 mg, Kawano et al. [10] reported that the mean Cmax was 14.7 μg/mL, and the mean trough concentration was 0.74 μg/mL. Aikawa et al. [12] described that the mean Cmax and trough concentration were 16.2 and 1.1 μg/mL, respectively. Sato et al. [4] performed PK-PD analysis involving 174 patients with MRSA infection. On logistic regression analysis, the efficacy was high when Cmax was 7.9–12.5 μg/mL (OR = 6.7), and the incidences of nephrotoxicity were 2.5, 5.2, and 13.

Symptomatic

patients diagnosed in childhood tend to have more severe disease manifestations [5], and are expected to experience an overall greater burden of disease [6] and [7]. selleck chemical Enzyme replacement therapy (ERT) is recommended for patients, including children, with GD who manifest signs and symptoms [6], [7] and [8]. Early intervention with ERT in symptomatic children may prevent the development of irreversible pathology [6], [7] and [8]. Treatment is also important to improve growth and reduce the impact of the disease on physical and psychosocial development [6], [7] and [8]. Taliglucerase alfa is an ERT that is approved in the United States, Israel, Brazil, Chile, Australia, Canada, and other countries for the treatment of Type 1 GD in adults, for treatment of pediatric patients in AZD0530 concentration the United States, Australia, and Canada, and for hematologic manifestations in pediatric patients with Type 3 GD in Canada. It is the first approved plant cell–expressed recombinant therapeutic protein [9]. Production in a plant cell culture system conveys potential advantages, such as the inability to be contaminated with or propagate mammalian pathogens, along with a potential lower cost [9], [10], [11] and [12]. In the taliglucerase alfa clinical development program, the phase 1 study

was conducted in 6 healthy adult volunteers [13]. Taliglucerase alfa safety and efficacy were then investigated in the phase 3, first-time-in-GD patients, pivotal, 9-month, double-blind, randomized, parallel-group trial in treatment-naïve adult patients [14]. Although it was not pre-specified in the trial

design, all 29 patients completing the study had Type 1 GD. Treatment with taliglucerase alfa 30 U/kg Idoxuridine and 60 U/kg (per infusion every other week) was associated with significant reductions in spleen volume, the primary end point, from baseline to 9 months. Secondary end points included significant reductions in liver volume and significant increases in hemoglobin concentrations and platelet counts from baseline to 9 months. Treatment was generally well tolerated and all drug-related adverse events (AEs) were mild/moderate and transient. The objective of this study was to assess the efficacy and safety of taliglucerase alfa in pediatric patients with GD at the same doses of 30 U/kg and 60 U/kg per infusion every other week as with the pivotal phase 3 trial in adult patients. This study was a phase 3B multicenter, randomized, double-blind, 2-dose trial of taliglucerase alfa (30 U/kg and 60 U/kg per infusion every other week) in pediatric patients (aged 2 to < 18 years). The trial was conducted at 3 centers (Shaare Zedek Medical Center, Jerusalem, Israel; Instituto Privado de Hematologia e Investigacion Clinica [I.P.H.I.C.], Asuncion, Paraguay; and the Morningside Medi-Clinic Johannesburg, South Africa).