Profile of idursulfase for the treatment of Hunter syndrome

Simona Sestito, Ferdinando Ceravolo, Michele Grisolia, Elisa Pascale, Licia Pensabene, Daniela Concolino Department of Pediatrics, University Magna Graecia of Catanzaro, Catanzaro, Italy Abstract: Mucopolysaccharidosis type II (MPS II; Hunter syndrome) is a rare X-linked lysosomal storage disorder caused by deficiency of the enzyme iduronate-2-sulfatase (IDS). Enzyme replacement therapy (ERT) with recombinant human IDS, available since 2005, is currently the most appropriate treatment for this progressive, multisystemic, chronic, and life-threatening disease. Efficacy and safety of therapy with idursulfase have been assessed in several clinical trials, and confirmed in many clinical reports. Long-term follow-up of patients receiving ERT has demonstrated the importance of an early onset of treatment with idursulfase, before irreversible pathological changes occur. Intravenously administered idursulfase is not able to cross the blood–brain barrier, so neurological signs and symptoms cannot benefit from ERT, still remaining a major challenge in the treatment of MPS II. Keywords: MPS II, glycosaminoglycans, enzyme replacement therapy, ER

Deuteron photo-disintegration with polarised photons in the energy range 30 - 50 MeV

The reaction d(\vec\gamma,np) has been studied using the tagged and polarised LADON gamma ray beam at an energy 30 - 50 MeV to investigate the existence of narrow dibaryonic resonances recently suggested from the experimental measurements in a different laboratory. The beam was obtained by Compton back-scattering of laser light on the electrons of the storage ring ADONE. Photo-neutron yields were measured at five neutron angle \vartheta_n = 22, 55.5, 90, 125 and 157 degrees in the center of mass system.Our results do not support the existence of such resonances.Comment: 16 pages, Latex, 22 figures, 1 table. Nucl. Phys. A to appea

Measuring star formation in high-z massive galaxies: A mid-infrared to submillimeter study of the GOODS NICMOS Survey sample

We present measurements of the mean mid-infrared-to-submillimeter flux densities of massive (M\ast \approx 2 \times 10^11 Msun) galaxies at redshifts 1.7 < z < 2.9, obtained by stacking positions of known objects taken from the GOODS NICMOS Survey (GNS) catalog on maps: at 24 {\mu}m (Spitzer/MIPS); 70, 100, and 160{\mu}m (Herschel/PACS); 250, 350, 500{\mu}m (BLAST); and 870{\mu}m (LABOCA). A modified blackbody spectrum fit to the stacked flux densities indicates a median [interquartile] star-formation rate of SFR = 63 [48, 81] Msun yr^-1 . We note that not properly accounting for correlations between bands when fitting stacked data can significantly bias the result. The galaxies are divided into two groups, disk-like and spheroid-like, according to their Sersic indices, n. We find evidence that most of the star formation is occurring in n \leq 2 (disk-like) galaxies, with median [interquartile] SFR = 122 [100,150] Msun yr^-1, while there are indications that the n > 2 (spheroid-like) population may be forming stars at a median [interquartile] SFR = 14 [9,20] Msun yr^-1, if at all. Finally, we show that star formation is a plausible mechanism for size evolution in this population as a whole, but find only marginal evidence that it is what drives the expansion of the spheroid-like galaxies.Comment: Accepted by MNRAS. 10 pages, 3 figures, 3 table

Combining the Hybrid Functional Method with Dynamical Mean-Field Theory

We present a new method to compute the electronic structure of correlated materials combining the hybrid functional method with the dynamical mean-field theory. As a test example of the method we study cerium sesquioxide, a strongly correlated Mott-band insulator. The hybrid functional part improves the magnitude of the pd-band gap which is underestimated in the standard approximations to density functional theory while the dynamical mean-field theory part splits the 4f-electron spectra into a lower and an upper Hubbard band.Comment: 5 pages, 2 figures, replaced with revised version, published in Europhys. Let

Indication to pelvic lymph nodes dissection for prostate cancer: the role of multiparametric magnetic resonance imaging when the risk of lymph nodes invasion according to Briganti updated nomogram is &lt;5

Background: The Briganti updated nomogram (BN) is the most popular predictive model aiming to predict the presence of lymph node invasion (LNI) in patients with prostate cancer (PCa), but it lacks information obtained by preoperative imaging. The primary aim of the study was to evaluate the role of multiparametric prostate magnetic resonance imaging (mp-MRI) in the indication to perform pelvic lymph nodes dissection (PLND) or not in patients with risk of LNI according to BN below 5%. Methods: Since March 2012 and September 2016, 310 patients who underwent a preoperative mp-MRI for staging purpose and subsequent robot-assisted extended PLND (RAEPLND) were retrospectively evaluated. Mp-MRIs were prospectively analyzed by two experienced radiologists. The imaging parameters analyzed were the presence of extracapsular extension (ECE), seminal vesicles invasion (SVI) and predominant Gleason pattern 4 (pG4). All patients underwent RAEPLND by two experienced surgeons with a standardized technique. A dedicated uropathologist performed all pathological analysis. Univariate analysis and multivariate logistic regression analysis were used in order to identify the predictors of LNI in patients with PCa. Results: In the overall population, 57 (18.4%) patients had histologically proven pN1 disease. 48/250 patients (19.2%) with a risk of LNI â¥5% as calculated by the BN were staged pN1 at final histopathological analysis. 9/60 patients (15.0%) with a risk of LNI <5% as calculated by BN, who underwent RAEPLND anyway according to the findings at mp-MRI, were staged pN1 at final histopathological analysis. At multivariate logistic regression analysis, all the three mp-MRI parameters were significant independent predictors of LNI after RAEPLND. Conclusions: The role of mp-MRI seemed to be crucial in patients with a risk of LNI <5% as calculated by the BN. The presence of ECE, SVI, or pG4 at mp-MRI was found to be an independent predictor of LNI by itself

Foregrounds in the BOOMERANG-LDB data: a preliminary rms analysis

We present a preliminary analysis of the BOOMERanG LDB maps, focused on foregrounds. BOOMERanG detects dust emission at moderately low galactic latitudes ($b > -20^o$) in bands centered at 90, 150, 240, 410 GHz. At higher Galactic latitudes, we use the BOOMERanG data to set conservative upper limits on the level of contamination at 90 and 150 GHz. We find that the mean square signal correlated with the IRAS/DIRBE dust template is less than 3% of the mean square signal due to CMB anisotropy