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9. Une couleur inhabituelle

Author

Campagne, J., primary, Kurt, S., additional, Moulinet, T., additional, Mohamed, S., additional, Deibener-Kaminsky, J., additional, and Jaussaud, R., additional

Published
2021
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13. Long-term benefit of enzyme replacement therapy with alglucosidase alfa in adults with Pompe disease: prospective analysis from the French Pompe Registry

Author

Semplicini, C., de Antonio, M., Taouagh, N., Béhin, A., Bouhour, F., Echaniz-Laguna, A., Magot, A., Nadaj-Pakleza, A., Orlikowski, D., Sacconi, S., Salort-Campana, E., Solé, G., Tard, Celine, Zagnoli, F., Jean-Yves, H., Hamroun, D., Laforêt, P., Attarian, S., Aubé-Nathier, A. C., Arrassi, A., Bassez, G., Bedat-Millet, A. L., Bouibede, F., Boyer, F. C., Caillaud, C., Canal, A., Carlier, R. Y., Chanson, J. B., Chapon, F., Cintas, P., Deibener-Kaminsky, J., Demurger, F., Desnuelle, C., Durieu, I., Eymard, B., Feasson, L., Fournier, M., Froissart, R., Furby, A., Garcia, P. Y., Germain, D. P., Ghorab, K., Morales, R. J., Krim, E., Labauge, P., Lacour, A., Lagrange, E., Lefeuvre, C., Leguy-Seguin, V., Leonard-Louis, S., Magy, L., Masseau, A., Michaud, M., Minot-Myhié, M. C., Nicolas, G., Nollet, S., Not, A., Noury, J. B., Ollivier, G., Péréon, Y., Perez, Thierry, Perniconi, B., Piraud, M., Petiot, P., Pouget, J., Praline, J., Prigent, H., Renard, D., Spinazzi, M., Stojkovic, T., Taithe, F., Tiffreau, Vincent, Vincent, D., Lille Neurosciences & Cognition - U 1172 (LilNCog), Institut National de la Santé et de la Recherche Médicale (INSERM)-Université de Lille-Centre Hospitalier Régional Universitaire [Lille] (CHRU Lille), Unité de Recherche Pluridisciplinaire Sport, Santé, Société (URePSSS) - ULR 7369 - ULR 4488 (URePSSS), and Université d'Artois (UA)-Université du Littoral Côte d'Opale (ULCO)-Université de Lille

Subjects
Adult, Male, Pediatrics, medicine.medical_specialty, Registry, alglucosidase alfa, Adolescent, [SDV]Life Sciences [q-bio], Walk Test, Disease, Sitting, 03 medical and health sciences, FEV1/FVC ratio, Young Adult, Glycogen storage disease type II, Genetics, medicine, Humans, Respiratory function, Prospective Studies, Registries, Child, Alglucosidase alfa, Genetics (clinical), 030304 developmental biology, Aged, 0303 health sciences, late onset Pompe disease, business.industry, 030305 genetics & heredity, alpha-Glucosidases, Enzyme replacement therapy, Middle Aged, medicine.disease, Respiratory Function Tests, Treatment Outcome, Ceiling effect, Female, France, business, medicine.drug, enzyme replacement therapy
Abstract

Despite a wide clinical spectrum, the adult form of Pompe disease is the most common one, and represents more than 90% of diagnosed patients in France. Since the marketing of enzyme replacement therapy (alglucosidase alfa, Myozyme), all reports to date in adults demonstrated an improvement of the walking distance, and a trend toward stabilization of respiratory function, but the majority of these studies were less than 5 years of duration. We report here the findings from 158 treated patients included in the French Pompe Registry, who underwent regular clinical assessments based on commonly used standardized tests (6-minute walking test, MFM scale, sitting vital capacity, MIP and MEP). For longitudinal analyses, the linear mixed effects models were used to assess trends in primary endpoints over time under ERT. A two-phase model better described the changes in distance traveled in the 6-minute walk test and MFM. 6MWT showed an initial significant increase (1.4% ± 0.5/year) followed by a progressive decline (-2.3%/year), with a cut-off point at 2.2 years. A similar pattern was observed in total MFM score (6.6% ± 2.3/year followed by a - 1.1%/year decline after 0.5 years). A single-phase decline with a slope of -0.9 ± 0.1%/year (P < .001) was observed for FVC, and MEP remained stable over the all duration of follow-up. This study provides further evidence that ERT improves walking abilities and likely stabilizes respiratory function in adult patients with Pompe disease, with a ceiling effect for the 6MWT in the first 3 years of treatment.

Published
2020

22. La Maladie de Duchenne au féminin

Author

Moulinet, T., primary, Campagne, J., additional, Mohamed, S., additional, Deibener-Kaminsky, J., additional, Guesdon, H., additional, Gomez, E., additional, and Jaussaud, R., additional

Published
2019
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28. Cross country maize market linkages in Africa: integration and price transmission across local and global markets

Author

Pierre, G. and Kaminsky, J.

Subjects
Marketing, FOS: Economics and business, Agricultural and Food Policy, Agricultural trade, Global VAR, Demand and Price Analysis, Price transmission, Market integration, Maize market
Abstract

In order to study short run price shock propagation, we model twenty seven sub-Saharan African domestic maize markets within a Global Vector AutorRegression framework. The main purpose is to fully embed multilateral trade flows as a way to better structure local price transmission dynamics and interdependencies and get a more comprehensive picture of food price shocks propagation. We found a generally weak integration of African domestic maize markets with regional and global markets. However, even in the absence of long run integration, between-country market contagion remains significant and short run price shocks propagate rapidly. Most local markets appear to be more responsive to local than to global shocks. We also identify price shock channels linking Western African countries to South Africa through maritime trade routes. Acknowledgement : This research was conducted in the context of Monitoring and Analyzing Food and Agricultural Policies (MAFAP) Program implemented by the Food and Agricultural Organization (FAO) in collaboration with the Organization for Economic Co-operation and Development (OECD) and financially supported by the Bill and Melinda Gates Foundation, USAID, the Netherlands and Germany. www.fao.org/in-action/mafap.

Published
2017
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29. Navigation assisted anterior screw fixation of a Type II dens axis fracture using a hybrid operating room

Author

Cink, V, Merkle, M, and Kaminsky, J

Subjects
dens fracture, ddc: 610, hybrid operating room, 610 Medical sciences, Medicine
Abstract

Objectives: Fractures of the Dens axis are often complex and a sufficient osteoynthesis may be challenging. The aim of this study was to prove the suitability of using a hybrid operating room and navigation Face-Mask-Referencing and Tracking device for anterior screw positioning in C2. Methods:[for full text, please go to the a.m. URL], Deutscher Kongress für Orthopädie und Unfallchirurgie (DKOU 2016)

Published
2016
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39. Myosites orbitaires : à propos d’une série de 22 patients

Author

Tholin, L., primary, Moulinet, T., additional, Revuz, S., additional, Campagne, J., additional, Bourne-Watrin, M., additional, Belan, M., additional, Mohamed, S., additional, Deibener-Kaminsky, J., additional, Maurier, F., additional, De Korwin, J.D., additional, Angioi-Duprez, K., additional, and Jaussaud, R., additional

Published
2018
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