Mucopolysaccharidosis type I (MPS I) is a rare lysosomal storage disorder treated with bone marrow transplantation or enzyme replacement therapy with laronidase, a high-cost orphan drug. Laronidase was approved by the US Food and Drug Administration and the European Medicines Agency in 2003 and by the Brazilian National Health Surveillance Agency in 2005. Many Brazilian MPS I patients have been receiving laronidase despite the absence of a governmental policy regulating access to the drug. Epidemiological and treatment data concerning MPS I (...) are scarce. This study aims to present a demographic profile of Brazilian patients with MPS I, describe the routes of access to laronidase in Brazil, and discuss associated ethical issues relating to public funding of orphan drugs. (shrink)

BackgroundPreprint usage is growing rapidly in the life sciences; however, questions remain on the relative quality of preprints when compared to published articles. An objective dimension of quality that is readily measurable is completeness of reporting, as transparency can improve the reader’s ability to independently interpret data and reproduce findings.MethodsIn this observational study, we initially compared independent samples of articles published in bioRxiv and in PubMed-indexed journals in 2016 using a quality of reporting questionnaire. After that, we performed paired comparisons (...) between preprints from bioRxiv to their own peer-reviewed versions in journals.ResultsPeer-reviewed articles had, on average, higher quality of reporting than preprints, although the difference was small, with absolute differences of 5.0% [95% CI 1.4, 8.6] and 4.7% [95% CI 2.4, 7.0] of reported items in the independent samples and paired sample comparison, respectively. There were larger differences favoring peer-reviewed articles in subjective ratings of how clearly titles and abstracts presented the main findings and how easy it was to locate relevant reporting information. Changes in reporting from preprints to peer-reviewed versions did not correlate with the impact factor of the publication venue or with the time lag from bioRxiv to journal publication.ConclusionsOur results suggest that, on average, publication in a peer-reviewed journal is associated with improvement in quality of reporting. They also show that quality of reporting in preprints in the life sciences is within a similar range as that of peer-reviewed articles, albeit slightly lower on average, supporting the idea that preprints should be considered valid scientific contributions. (shrink)

Background: Mental healthcare needs person-tailored interventions. Experience Sampling Method (ESM) can provide daily life monitoring of personal experiences. This study aims to operationalize and test a measure of momentary reward-related Quality of Life (rQoL). Intuitively, quality of life improves by spending more time on rewarding experiences. ESM clinical interventions can use this information to coach patients to find a realistic, optimal balance of positive experiences (maximize reward) in daily life. rQoL combines the frequency of engaging in a relevant context (a (...) ‘behavior setting’) with concurrent (positive) affect. High rQoL occurs when the most frequent behavior settings are combined with positive affect or infrequent behavior settings co-occur with low positive affect. Methods: Resampling procedures (Monte Carlo experiments) were applied to assess the reliability of rQoL using various behavior setting definitions under different sampling circumstances, for real or virtual subjects with low-, average- and high contextual variability. Furthermore, resampling was used to assess whether rQoL is a distinct concept from positive affect. Virtual ESM beep datasets were extracted from 1058 valid ESM observations for virtual and real subjects. Results: Behavior settings defined by Who-What contextual information were most informative. Simulations of at least 100 ESM observations are needed for reliable assessment. Virtual ESM beep datasets of a real subject can be defined by Who-What-Where behavior setting combinations. Large sample sizes are necessary for reliable rQoL assessments, except for subjects with low contextual variability. rQoL is distinct from positive affect. Conclusion: rQoL is a feasible concept. Monte Carlo experiments should be used to assess the reliable implementation of an ESM statistic. Future research in ESM should asses the behavior of summary statistics under different sampling situations. This exploration is especially relevant in clinical implementation, where often only small datasets are available. (shrink)

Starting from the first centenary of Carlo Cattaneo's birth in 1901, the Author reviews the publication of his works in concomitance with the interpretation and diffusion of his thinking. From the first ample collection of writings, down to Bertani and other friends, to the anthology by Gaetano Salvemini, from Alessandro Levi's monograph to the interpretations of Norberto Bobbio and Luigi Ambrosoli, the article shows the interweaving between the exegesis and the piecing together of Cattaneo's dispersed works, the complete retrieval of (...) which is being undertaken by the Italo-Swiss Committee set up in 1946. It ends with the new national edition of his works, which on the occasion of the 150th anniversary of his death in 2019 saw the publication of the fourth volume of his Lettere, the eighth volume in the entire collection. (shrink)

In this paper, we present one of the main starting points of naturalism in ethics: Geach’s challenge against non-cognitivism. We try to find an answer to Geach’s challenge in the notion of family resemblance applied to ethics. In doing so we recover a not much-discussed influence of Moore on Wittgenstein’s conception of family resemblance, which leads us to define Wittgenstein as non-non-cognitivist in ethics. -/- Pre print (some changes in the published edition).

In this paper I will discuss Edmund Husserl’s critique of Franz Brentano’s interpretation of categorical judgments as Double Judgments (Doppelurteile). This will be developed mostly as an internal critique, within the framework of the school of Brentano, and not through a direct contrast with Husserl’s own theory of judgment, as presented e.g. in the Fifth Investigation. Already during the 1890s Husserl overcame the psychologistic aspects of Brentano’s approach, advocating the importance of analysing the logical structure underlying language independently from psychology. (...) Moreover, Husserl’s critique seems to be also applicable to Bertrand Russell’s analysis, which shares an important aspect of Brentano’s theory. I will try to avoid going too deep into the various theories of judgment and keep mostly to the issue of double judgments. (shrink)

Ricordo di Carlo Ascheri, di F. Cerutti.--Note sulla vita di Giovanni Francesco Sannazari della Ripa (1480c.-1535) fino al lettorato avignonese, di M. Ascheri.--Noterelle machiavelliane, di N. Badaloni.--Un argumento consolatorio dell'Assioco nella tradizione platonica, di A. Cardini.--Zeller i Feuerbach, di C. Cesa.-- Ludwig Feuerbach in den Buberschen Sicht, di I. Dubský.--Lo Short tract on first principles di Hobbes e la metafisica della luce di Roberto Grossatesta, di A.G. Gargani.--Un letterato e una strega al principio dell'500: Panfilo Sasso e Anastasia la Fraappona, (...) di C. Ginzburg. (shrink)