The TREAT-NMD Duchenne Muscular Dystrophy Registries: Conception, Design, and Utilization by Industry and Academia.

Duchenne muscular dystrophy (DMD) is an X-linked genetic disease, caused by the absence of the dystrophin protein. Although many novel therapies are under development for DMD, there is currently no cure and affected individuals are often confined to a wheelchair by their teens and die in their twenties/thirties. DMD is a rare disease (prevalence <5/10,000). Even the largest countries do not have enough affected patients to rigorously assess novel therapies, unravel genetic complexities, and determine patient outcomes. TREAT-NMD is a worldwide network for neuromuscular diseases that provides an infrastructure to support the delivery of promising new therapies for patients. The harmonized implementation of national and ultimately global patient registries has been central to the success of TREAT-NMD. For the DMD registries within TREAT-NMD, individual countries have chosen to collect patient information in the form of standardized patient registries to increase the overall patient population on which clinical outcomes and new technologies can be assessed. The registries comprise more than 13,500 patients from 31 different countries. Here, we describe how the TREAT-NMD national patient registries for DMD were established. We look at their continued growth and assess how successful they have been at fostering collaboration between academia, patient organizations, and industry.

University of Iowa John Pappajohn Entrepreneurial Center annual report, FY 2011.

Annual report of the John Pappajohn Entrepreneurial Center at the University of Iowa.

University of Iowa John Pappajohn Entrepreneurial Center annual report, FY 2012.

Annual report of the John Pappajohn Entrepreneurial Center at the University of Iowa.

University of Iowa John Pappajohn Entrepreneurial Center annual report, FY 2013.

Annual report of the John Pappajohn Entrepreneurial Center at the University of Iowa.

Unlicensed and off-label drug use in a Swiss paediatric university hospital.

BACKGROUND: Many medicines used in newborns, infants, children and adolescents are not licensed ("unlicensed") or are prescribed outside the terms of the marketing authorization ("off-label"). Several studies have shown that this is a common practice in various healthcare settings in the USA, Europe and Australia, but data are scarce in Switzerland. OBJECTIVES: The aim of our prospective study was to determine the proportion of unlicensed or off-label prescriptions in paediatric patients. METHODS: This pilot study was conducted prospectively over a six month period in the department of paediatrics of a university hospital. RESULTS: Sixty patients aged from three days to 14 years were included in the study. A total of 483 prescriptions were written for the patients. More than half of all prescriptions (247; 51%) followed the terms of the marketing authorization. 114 (24%) were unlicensed and 122 (25%) off-label. All patients received at least one unlicensed or offlabel medicine. CONCLUSION: The use of unlicensed or off-label medicines to treat children was found to be common. Co-operation between the pharmaceutical industry, national regulatory authorities, clinical researchers, healthcare professionals and parents is required in order to ensure that children do not remain "therapeutic orphans".

Electronic Construction Collaboration - Phase 1, 2009

Bridge construction projects are becoming increasingly complex as the demand for context-sensitive solutions, aesthetic designs, and accelerated bridge construction becomes more prevalent. In addition, the Iowa Department of Transportation (Iowa DOT) is entering a phase of design and construction of large border bridges, such as the I-80 (let 2008 for $56 million) and US 34 bridges over the Missouri River and I-74 over the Mississippi River. Compared to typical construction projects, these bridges generate more contractor Requests for Information (RFIs), Value Engineering (VE) proposals, Requests for Changes (RFCs), and shop drawings. Management of these submittals is a significant challenge for Resident Construction Engineers (RCEs) and other Iowa DOT staff. In addition, some submittals require cross-departmental and project consultant reviews. Commercially available software exists for managing submittals and project collaboration teams; in-house solutions may also be possible. Implementation is intended to speed construction submittal review time, reduce incidence of delay claims, and free up Iowa DOT staff from project management administrative tasks. Researchers from Iowa State University working with the Iowa DOT conducted a multi-pronged approach to indentify a web-based collaboration solution for Iowa DOT bridge projects. An investigation was launched to determine the functional needs of the Iowa DOT. Commercially available software programs were also evaluated to find what functionality is currently available. A Request for Proposals (RFP) was written to select a commercial web-based collaboration solution for pilot testing. In the second phase of research, a solution will be selected and implemented on two pilot projects. Lessons learned from these pilot projects will assist the Iowa DOT in developing and implementing a long-term solution to improve the management of Iowa DOT bridge projects.

A course on collaboration between social workers and general practitioners during their vocational training

The educational programme reported was an experiment in the vocational training scheme of the department of General Practice, Erasmus University, Rotterdam, Holland, and is now part of the course. The programme focused on the training in team function (co-operation) given to trainee GPs and social workers. It became clear that both groups during their professional training develop markedly different attitudes and views about patient (client) care. These differences form a fundamental handicap in any discussion about teamwork. During the programme the students were made aware of this divergence of viewpoint and were taught how to handle these resulting handicaps and, if possible, to eliminate them.

Timing of Capacity Change: Models for Capital Intensive Industry

The suitable timing of capacity investments is a remarkable issue especially in capital intensive industries. Despite its importance, fairly few studies have been published on the topic. In the present study models for the timing of capacity change in capital intensive industry are developed. The study considers mainly the optimal timing of single capacity changes. The review of earlier research describes connections between cost, capacity and timing literature, and empirical examples are used to describe the starting point of the study and to test the developed models. The study includes four models, which describe the timing question from different perspectives. The first model, which minimizes unit costs, has been built for capacity expansion and replacement situations. It is shown that the optimal timing of an investment can be presented with the capacity and cost advantage ratios. After the unit cost minimization model the view is extended to the direction of profit maximization. The second model states that early investments are preferable if the change of fixed costs is small compared to the change of the contribution margin. The third model is a numerical discounted cash flow model, which emphasizes the roles of start-up time, capacity utilization rate and value of waiting as drivers of the profitable timing of a project. The last model expands the view from project level to company level and connects the flexibility of assets and cost structures to the timing problem. The main results of the research are the solutions of the models and analysis or simulations done with the models. The relevance and applicability of the results are verified by evaluating the logic of the models and by numerical cases.

Purification and fractionation by nanotiltration in dairy and sugar and sweetener industry applications

Nanotiltration is a membrane separation method known for its special characteristic of rejecting multivalent ions and passing monovalent ions. Thus, it is commonly applied with dilute aqueous solutions in partial salt removal, like in drinking water production. The possibilities of nanofiltration have been studied and the technique applied in a wide branch of industries, e.g. the pulp and paper, the textile and the chemical processing industry. However, most present applications and most of the potential applications studied involve dilute solutions, the permeating stream being generally water containing monovalent salts. In this study nanotiltration is investigated more as a fractionation method. A well-known application in the dairy industry is concentration and partial salt removal from whey. Concentration and partial demineralization is beneficial for futher processing of whey as whey concentrates are used e.g. in baby foods. In the experiments of this study nanotiltration effectively reduced the monovalent salts in the whey concentrate. The main concern in this application is lactose leakage into the permeate. With the nanofiltration membranes used the lactose retentions were practically ? 99%. Another dairy application studied was the purification and reuse of cleaning solutions. This is an environmentally driven application. An 80% COD reduction by nanofiltration was observed for alkaline cleaning-in-place solution. Nanofiltration is not as commonly applied in the sugar and sweeteners industry as in the dairy industry. In this study one potential application was investigated, namely xylose purification from hemicellulose hydrolyzate. Xylose is raw material for xylitol production. Xylose separation from glucose was initially studied with xylose-glucose model solutions. The ability of nanofiltration to partially separate xylose into the permeate from rather concentrated xylose-glucose solutions (10 w-% and 30 w-%) became evident. The difference in size between xylose and glucose molecules according to any size measure is small, e.g. the Stokes diameter of glucose is 0.73 nm compared to 0.65 nm for xylose. In further experiments, xylose was purified into nanoliltration permeate from a hemicellulose hydrolyzate solution. The xylose content in the total solids was increased by 1.4—1.7 fold depending on temperature, pressure and feed composition.

Multidisciplinary practice vs. inter-firm collaboration in professional services market

Yritysten välinen yhteistyö kasvaa asiantuntijamarkkinoilla. Suppean palvelutarjooman omaavat yritykset muodostavat laajempia palveluita yhdistämällä osaamisiaan kumppaneidensa kanssa. Näin muodostuvat yritysryhmittymät uhkaavat alaa hallitsevia monipuolisen palvelutarjooman omaavia kansainvälisiä moniosaajayrityksiä. Tämän diplomityön tavoitteena on selvittää minkälaisia hyötyjä moniosaajayritys voi saada näitä ryhmittymiä vastaan lisäämällä omaa yhteistyötään. Tavoitteeseen pääsemiseksi markkinoilla olevat yritysryhmittymät tunnistetaan ja selvitetään minkälaisia asioita asiakas pitää tärkeänä ostaessaan asiantuntijapalveluita. Toimialan trendit ja aikaisemmat tutkimukset yritysten välisestä yhteistyöstä sekä asiakkaan ostokäyttäytymisestä osoittavat, että yhteistyön avulla yrityksellä on mahdollisuus saavuttaa monia hyötyjä. Tietoa olemassa olevista yritysryhmittymistä ja asiakkaiden ostokäyttäytymisestä kerättiin haastattelemalla yhden kansainvälisen moniosaajayrityksen henkilöstöä sekä asiakkaita. Tuloksena löytyi yritysryhmittymiä, joista osa uhkaa moniosaajayrityksen kilpailuetua. Asiakkaiden ostokäyttäyminen suosi hieman enemmän asiantuntijapalveluiden hankkimista yritysryhmittymältä moniosaajayrityksen sijaan. Tekemällä yhteistyötä ja tarjoamalla tiettyjä palveluita yhdessä kumppanin kanssa, moniosaajayritys voi saavuttaa hyötyjä yritysryhmittymiä vastaan ja vaikuttaa positiivisesti asiakkaan ostokäyttäytymiseen.

Are Evaluation of ICTs Investment Using Decision Support Systems:Case Applications from Distributor's and End User's Perspective Group Decision

The evaluation of investments in advanced technology is one of the most important decision making tasks. The importance is even more pronounced considering the huge budget concerning the strategic, economic and analytic justification in order to shorten design and development time. Choosing the most appropriate technology requires an accurate and reliable system that can lead the decision makers to obtain such a complicated task. Currently, several Information and Communication Technologies (ICTs) manufacturers that design global products are seeking local firms to act as their sales and services representatives (called distributors) to the end user. At the same time, the end user or customer is also searching for the best possible deal for their investment in ICT's projects. Therefore, the objective of this research is to present a holistic decision support system to assist the decision maker in Small and Medium Enterprises (SMEs) - working either as individual decision makers or in a group - in the evaluation of the investment to become an ICT's distributor or an ICT's end user. The model is composed of the Delphi/MAH (Maximising Agreement Heuristic) Analysis, a well-known quantitative method in Group Support System (GSS), which is applied to gather the average ranking data from amongst Decision Makers (DMs). After that the Analytic Network Process (ANP) analysis is brought in to analyse holistically: it performs quantitative and qualitative analysis simultaneously. The illustrative data are obtained from industrial entrepreneurs by using the Group Support System (GSS) laboratory facilities at Lappeenranta University of Technology, Finland and in Thailand. The result of the research, which is currently implemented in Thailand, can provide benefits to the industry in the evaluation of becoming an ICT's distributor or an ICT's end user, particularly in the assessment of the Enterprise Resource Planning (ERP) programme. After the model is put to test with an in-depth collaboration with industrial entrepreneurs in Finland and Thailand, the sensitivity analysis is also performed to validate the robustness of the model. The contribution of this research is in developing a new approach and the Delphi/MAH software to obtain an analysis of the value of becoming an ERP distributor or end user that is flexible and applicable to entrepreneurs, who are looking for the most appropriate investment to become an ERP distributor or end user. The main advantage of this research over others is that the model can deliver the value of becoming an ERP distributor or end user in a single number which makes it easier for DMs to choose the most appropriate ERP vendor. The associated advantage is that the model can include qualitative data as well as quantitative data, as the results from using quantitative data alone can be misleading and inadequate. There is a need to utilise quantitative and qualitative analysis together, as can be seen from the case studies.

ICT-alan yrittäjyyden alueellinen edistäminen CASE: Etelä-Karjala

Tutkimuksen tarkoituksena oli selvittää syy siihen, miksi Etelä-Karjalassa syntyy vähemmän uusia ICT-alan yrityksiä, kuin mihin on edellytyksiä. Tutkimusmetodologia oli toiminta-analyyttinen ja sen empiirinen osuus muodostettiin avoimien teemahaastatteluiden avulla. Haastatellut olivat alueen ICTalan päättäjiä ja heitä oli yhteensä 28. Merkittävimpinä syinä siihen, että korkeakoulun, ammattikorkeakoulun ja teknologiakeskuksen olemassaolosta huolimatta maakuntaan syntyy varsin vähän uusia ICT-alan yrityksiä, nähtiin olevan negatiivinen ilmapiiri ja kateus yrittäjyyttä kohtaan, lähimarkkinoiden pieni koko sekä maakunnallisen yhteistyön puuttuminen ICT-alalla. Kehittämisalueina nousivat esiin koulutus, alueellinen yhteistyö, yritysyhteistyö, infrastruktuurin kehittäminen, tutkimus- ja tuotekehitys sekä alueen markkinointi. Ohjeeksi ICT-alan yrittäjyyden edistämiseksi Etelä-Karjalassa tutkimuksessa annettiin laajan yhteisen tahdon aikaansaaminen ja resurssien kohdentaminen jollekin valitulle ICT-alan osa-alueelle tavoitteena synnyttää alueelle kansainvälisesti merkittävä osaamiskeskittymä.

Market Orientation in Rapidly Internationalizing Small Companies — Evidence from the Software Industry

For more than a decade, researchers have been aware of the increased pace of small-firm internationalization and the greater effect of these rapidly growing small businesses on the wealth, international trade, and job-creation opportunities of countries. Due to the small size of the home market, Finnish companies have been generally considered highly interested in internationalization. One particular domain in which rapid internationalization has been considered feasible is the global software business, with its knowledge-intensive nature and high growth potential. However, over time the failure rate of small entrepreneurial firms has remained especially high in high-technology markets. One of the reasons for this seems to lie in the fact that these companies are often formed by people with a strong technological background but limited competences in other areas. Further, research on the marketing capabilities of rapidly internationalizing high-tech firms has been scarce thus far. In addition, while there is much research on the first years of operations of rapidly internationalizing companies, it is not well known what becomes of them later on. Therefore, there is a need for more investigation into the managerial mindset, competences and decision-making in these small companies, especially from the perspective of how they acquire and exploit market knowledge, and enhance their networking capabilities in order to promote international expansion. The present study focuses on market orientation in small software firms that internationalize their operations rapidly in global software markets. It builds on qualitative data to illustrate how these companies develop their market-oriented product-market strategies during the process of increasing international commitment. It also shows how they manage their network relationships in order to be able to offer better customer service and to thrive in the fierce global competition. The study was conducted in the empirical context of Finnish small software companies, and the main data consists of interviews with top managers in these businesses. The interviews were designed to cover a minimum period of five years of the company's international operations, thus offering a retrospective in-depth perspective on market orientation, internationalization and partnerships in the given context. One particular focus is on less successfully internationalized software companies, and the challenges they face when approaching international markets. This study makes a significant contribution to the literature on market orientation for several reasons. First, building on data from the software industry, it clarifies the existing theory in the context of rapid internationalization and network relationships. Secondly, it provides a good body of evidence on market orientation in both successfully and less successfully internationalized companies, and identifies the key related differences between the two company groups. Thirdly, it highlights the importance of inter-firm networks in the rapid internationalization of small software firms, providing companies with important market knowledge and, in some cases, management challenges. Fourthly, this investigation clarifies market orientation in the context of different software-product strategies, thus, combining the perspectives of market orientation in both manufacturing and services. In sum, the results of the study are significant for both small software firms and public-policy makers since they shed light on the market-oriented managerial mindset and the market-information gathering and sharing processes that are needed in successful rapid internationalization.

La collaboration interprofessionnelle à l'interface des institutions socio-éducative et psychiatrique. Jeux et enjeux de l'intervention auprès de personnes en situation de handicap mental et de troubles psychiques

La prise en charge et le suivi de personnes en situation de handicap mental souffrant de troubles psychiques et se trouvant donc à l'interface des domaines socio:éducatif et psychiatrique, constituent des défis complexes en matière de collaboration interprofessionnelle. Dans le canton de Vaud, les acteurs concernés par ce problème s'efforcent depuis de nombreuses années de créer des réseaux pluridisciplinaires visant un meilleur échange entre professionnels et le développement de compétences et de connaissances permettant d'améliorer le bien:être des bénéficiaires. Ce travail se propose ainsi d'étudier et de questionner ces modalités de travail dans une perspective socioculturelle (Vygotski, 1934/1997), afin d'en comprendre le fonctionnement, d'en éclairer les mécanismes et de fournir des pistes de réflexion aux professionnels. Il repose sur un travail de terrain mené auprès des membres du Dispositif de Collaboration Psychiatrie Handicap Mental (DCPHM) du Département de psychiatrie du CHUV, dont la mission principale est de faciliter la collaboration entre les institutions socio:éducatives et psychiatriques spécialisées dans le suivi des personnes en situation de handicap mental et souffrant de troubles psychiques. Le travail empirique est basé sur une approche qualitative et compréhensive des interactions sociales, et procède par une étude de terrain approfondie. Les données recueillies sont variées : notes de terrain et récolte de documentation, enregistrement de réunions d'équipe au sein du DCPHM et de réunions de réseau, et entretiens de différents types. L'analyse montre que le travail de collaboration qui incombe à l'équipe est constitué d'obstacles qui sont autant d'occasions de développement professionnel et de construction identitaire. Les résultats mettent en lumière des mécanismes discursifs de catégorisation concourant à la fois à la construction des patients comme objets d'activité, et à la construction d'une place qui légitime les interventions de l'équipe dans le paysage socio:éducatif et psychiatrique vaudois et la met au centre de l'arène professionnelle. -- Care and follow:up for people with mental disabilities suffering from psychological disorders : therefore at the interface between the socio:educational and psychiatric fields : represent complex challenges in terms of interprofessional collaboration. In the canton of Vaud, the caregivers involved in this issue have been trying for years to build multidisciplinary networks in order to better exchange between professionals and develop skills and knowledge to improve the recipients' well:being. This work thus proposes to study and question these working methods in a sociocultural perspective (Vygotski, 1934/1997) so as to understand how they operate, highlight inherent mechanisms and provide actionable insights to the professionals. It is based on fieldwork conducted among members of the Dispositif de Collaboration Psychiatrie Handicap Mental (DCPHM), of the Psychiatry Department at the CHUV University Hospital in Lausanne, whose main mission is to facilitate collaboration between the socio:educational and psychiatric institutions specialising in monitoring people presenting with both mental handicap and psychiatric disorder. The empirical work is based on a qualitative and comprehensive approach to social interactions, and conducted based on an in:depth field study. The data collected are varied - field notes and documentation collection, recordings of team meetings within the DCPHM and network meetings, and various types of interviews. The analysis shows that the collaborative work that befalls the team consists of obstacles, all of which provide opportunities for professional development and identity construction. The results highlight discursive strategies of categorisation which contribute both to the construction of the patients as objects of activity and to building a position that legitimates the team's interventions in the socio: educational and psychiatric landscape of canton Vaud and puts it in the centre of the professional arena.

Clustering and international competitiveness of information technology industry in the Saint Petersburg area

The main objective of this study is to assess the potential of the information technology industry in the Saint Petersburg area to become one of the new key industries in the Russian economy. To achieve this objective, the study analyzes especially the international competitiveness of the industry and the conditions for clustering. Russia is currently heavily dependent on its natural resources, which are the main source of its recent economic growth. In order to achieve good long-term economic performance, Russia needs diversification in its well-performing industries in addition to the ones operating in the field of natural resources. The Russian government has acknowledged this and started special initiatives to promote such other industries as information technology and nanotechnology. An interesting industry that is basically less than 20 years old and fast growing in Russia, is information technology. Information technology activities and markets are mainly concentrated in Russia’s two biggest cities, Moscow and Saint Petersburg, and areas around them. The information technology industry in the Saint Petersburg area, although smaller than Moscow, is especially dynamic and is gaining increasing foreign company presence. However, the industry is not yet internationally competitive as it lacks substantial and sustainable competitive advantages. The industry is also merely a potential global information technology cluster, as it lacks the competitive edge and a wide supplier and manufacturing base and other related parts of the whole information technology value system. Alone, the industry will not become a key industry in Russia, but it will, on the other hand, have an important supporting role for the development of other industries. The information technology market in the Saint Petersburg area is already large and if more tightly integrated to Moscow, they will together form a huge and still growing market sufficient for most companies operating in Russia currently and in the future. Therefore, the potential of information technology inside Russia is immense.