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19 results on '"de Lattre, Capucine"'

3. Myostatin: a Circulating Biomarker Correlating with Disease in Myotubular Myopathy Mice and Patients

Author

Annoussamy, Mélanie, Seferian, Andreea, Baets, Jonathan, Voermans, Nicole, Behin, Antony, Schara, U., D’Amico, Adele, Hernandez, Arturo, de Lattre, Capucine, Arnal, Jean-Michel, Mayer, Michèle, Cuisset, Jean-Marie, Vuillerot, Carole, Fontaine, Stéphanie, Bellance, Rémy, Koch, Catherine, Buono, Suzie, Menuet, Alexia, Robé, Anne, Djeddi, Sarah, Kretz, Christine, Gomez-Oca, Raquel, Depla, Marion, Monseur, Arnaud, Thielemans, Leen, Servais, Laurent, Laporte, Jocelyn, and Cowling, Belinda S.

Published
2020
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4. X-linked myotubular myopathy: A prospective international natural history study

Author

Annoussamy, Mélanie, Lilien, Charlotte, Gidaro, Teresa, Gargaun, Elena, Chê, Virginie, Schara, Ulrike, Gangfuß, Andrea, DʼAmico, Adele, Dowling, James J., Darras, Basil T., Daron, Aurore, Hernandez, Arturo, de Lattre, Capucine, Arnal, Jean-Michel, Mayer, Michèle, Cuisset, Jean-Marie, Vuillerot, Carole, Fontaine, Stéphanie, Bellance, Rémi, Biancalana, Valérie, Buj-Bello, Ana, Hogrel, Jean-Yves., Landy, Hal, and Servais, Laurent

Published
2019
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5. Parent satisfaction with medical and rehabilitation services for children with physical disabilities during lockdown

Author

Cacioppo, Marine, primary, Lucas, Clémence, additional, Dai, Shenhao, additional, Bailly, Rodolphe, additional, Pérennou, Dominic, additional, Varengue, Roxane, additional, Houx, Laetitia, additional, Lempereur, Mathieu, additional, Kandalaft, Christèle, additional, Chatelin, Alain, additional, Vagnoni, Jacky, additional, Vuillerot, Carole, additional, Gautheron, Vincent, additional, Dinomais, Mickael, additional, Dheilly, Elea, additional, Bouvier, Sandra, additional, Brochard, Sylvain, additional, Pons, Christelle, additional, Filly, Emmanuelle, additional, Mensah-Gourmel, Johanne, additional, Génot, Amélie, additional, Ropars, Juliette, additional, Toullet, Philippe, additional, De Lattre, Capucine, additional, Klinger, Evelyne, additional, Laffont, Isabelle, additional, Sitruk, Clara, additional, Van Boagert, Patrick, additional, and Tessiot, Chantal, additional

Published
2023
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6. Correction to: Use of MFM-20 to monitor SMA types 1 and 2 patients treated with nusinersen

Author

Le Goff, Laure, primary, Seferian, Andreea, additional, Phelep, Aurelie, additional, Rippert, Pascal, additional, Mathieu, Marie-Laure, additional, Cances, Claude, additional, de Lattre, Capucine, additional, Durigneux, Julien, additional, Gousse, Gaelle, additional, Vincent-Genod, Dominique, additional, Ribault, Shams, additional, Gómez García de la Banda, Marta, additional, Quijano-Roy, Susana, additional, Sarret, Catherine, additional, Servais, Laurent, additional, and Vuillerot, Carole, additional

Published
2022
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7. Parent satisfaction with medical and rehabilitation services for children with physical disabilities during lockdown

Author

Filly, Emmanuelle, Mensah-Gourmel, Johanne, Génot, Amélie, Ropars, Juliette, Toullet, Philippe, De Lattre, Capucine, Klinger, Evelyne, Laffont, Isabelle, Sitruk, Clara, Van Boagert, Patrick, Tessiot, Chantal, Cacioppo, Marine, Lucas, Clémence, Dai, Shenhao, Bailly, Rodolphe, Pérennou, Dominic, Varengue, Roxane, Houx, Laetitia, Lempereur, Mathieu, Kandalaft, Christèle, Chatelin, Alain, Vagnoni, Jacky, Vuillerot, Carole, Gautheron, Vincent, Dinomais, Mickael, Dheilly, Elea, Bouvier, Sandra, Brochard, Sylvain, and Pons, Christelle

Published
2023
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8. Evidence-Based, Implementable Motor Rehabilitation Guidelines for Individuals With Cerebral Palsy

Author

Demont, Anthony, primary, Gedda, Michel, additional, Lager, Céline, additional, de Lattre, Capucine, additional, Gary, Yann, additional, Keroulle, Elisabeth, additional, Feuillerat, Brigitte, additional, Caudan, Hervé, additional, Sancelme, Zoé, additional, Isapof, Arnaud, additional, Viehweger, Elke, additional, Chatelin, Matthieu, additional, Hochard, Marianne, additional, Boivin, Julia, additional, Vurpillat, Pascale, additional, Genès, Nathalie, additional, de Boissezon, Xavier, additional, Fontaine, Audrey, additional, and Brochard, Sylvain, additional

Published
2022
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10. Motor and respiratory heterogeneity in Duchenne patients: Implication for clinical trials

Author

Humbertclaude, Véronique, Hamroun, Dalil, Bezzou, Kamel, Bérard, Carole, Boespflug-Tanguy, Odile, Bommelaer, Christine, Campana-Salort, Emmanuelle, Cances, Claude, Chabrol, Brigitte, Commare, Marie-Christine, Cuisset, Jean-Marie, de Lattre, Capucine, Desnuelle, Claude, Echenne, Bernard, Halbert, Cécile, Jonquet, Olivier, Labarre-Vila, Annick, N’Guyen-Morel, Marie-Ange, Pages, Michel, Pepin, Jean-Louis, Petitjean, Thierry, Pouget, Jean, Ollagnon-Roman, Elisabeth, Richelme, Christian, Rivier, François, Sacconi, Sabrina, Tiffreau, Vincent, Vuillerot, Carole, Picot, Marie-Christine, Claustres, Mireille, Béroud, Christophe, and Tuffery-Giraud, Sylvie

Published
2012
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11. Leveraging Natural History Data in One- and Two-Arm Hierarchical Bayesian Studies of Rare Disease Progression.

Author

Monseur, Arnaud, Carlin, Bradley P., Boulanger, Bruno, Seferian, Andreea, Servais, Laurent, Freitag, Chris, Thielemans, Leen, the NatHis-MTM Study Group, Gidaro, Teresa, Gargaun, Elena, Chê, Virginie, Schara, Ulrike, Gangfuß, Andrea, D'Amico, Adele, Dowling, James J., Darras, Basil T., Daron, Aurore, Hernandez, Arturo, de Lattre, Capucine, and Arnal, Jean-Michel

Abstract

The small sample sizes inherent in rare and pediatric disease settings offer significant challenges for clinical trial design. In such settings, Bayesian adaptive trial methods can often pay dividends, allowing the sensible incorporation of auxiliary data and other relevant information to bolster that collected by the trial itself. Previous work has also included the use of one-arm trials augmented by the participants' own natural history data, from which the future course of the disease in the absence of intervention can be predicted. Patient response can then be defined by the degree to which post-intervention observations are inconsistent with the predicted "natural" trajectory. While such trials offer obvious advantages in efficiency and ethical hazard (since they expose no new patients to a placebo, anathema to patients or their parents and caregivers), they can offer no protection against bias arising from the presence of any "placebo effect," the tendency of patients to improve merely by being in the trial. In this paper, we investigate the impact of both static and transient placebo effects on one-arm responder studies of this type, as well as two-arm versions that incorporate a small concurrent placebo group but still borrow strength from the natural history data. We also propose more traditional Bayesian changepoint models that specify a parametric functional form for the patient's post-intervention trajectory, which in turn allow quantification of the treatment benefit in terms of the model parameters, rather than semi-parametrically in terms of a response relative to some "null" model. We compare the operating characteristics of our designs in the context of an ongoing investigation of centronuclear myopathies (CNMs), a group of congenital neuromuscular diseases whose most common and severe form is X-linked, affecting approximately 1 in 50,000 newborn boys. Our results indicate our two-arm responder and changepoint methods can offer protection against placebo effects, improving power while protecting the trial's Type I error rate. However, further research into innovative trial designs as well as ongoing dialog with regulatory authorities remain critically important in rare disease research. [ABSTRACT FROM AUTHOR]

Published
2022
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12. Myostatin: a Circulating Biomarker Correlating with Disease in Myotubular Myopathy Mice and Patients

Author

Koch, Catherine, primary, Buono, Suzie, additional, Menuet, Alexia, additional, Robé, Anne, additional, Djeddi, Sarah, additional, Kretz, Christine, additional, Gomez-Oca, Raquel, additional, Depla, Marion, additional, Monseur, Arnaud, additional, Thielemans, Leen, additional, Servais, Laurent, additional, Laporte, Jocelyn, additional, Cowling, Belinda S., additional, Annoussamy, Mélanie, additional, Seferian, Andreea, additional, Baets, Jonathan, additional, Voermans, Nicole, additional, Behin, Antony, additional, Schara, U., additional, D’Amico, Adele, additional, Hernandez, Arturo, additional, de Lattre, Capucine, additional, Arnal, Jean-Michel, additional, Mayer, Michèle, additional, Cuisset, Jean-Marie, additional, Vuillerot, Carole, additional, Fontaine, Stéphanie, additional, and Bellance, Rémy, additional

Published
2020
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18. Multidimensional Outcome Assessment in Cerebral Palsy

Author

Viehweger, Elke, primary, Haumont, Thierry, additional, de Lattre, Capucine, additional, Presedo, Ana, additional, Filipetti, Paul, additional, Ilharreborde, Brice, additional, Lebarbier, Pierre, additional, Loundou, Anderson, additional, and Simeoni, Marie-Claude, additional

Published
2008
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