Search Results - "Gusset, Nicole"

The need for evidence‐based treatment decisions in spinal muscular atrophy type 0 by Erbas, Yasemin, Gusset, Nicole

“…Importantly, as with other SMA types, within the SMA type 0 subpopulation there is evidence of heterogeneity in both disease severity and responsiveness to…”
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A Patient-Centered Evaluation of Meaningful Change on the 32-Item Motor Function Measure in Spinal Muscular Atrophy Using Qualitative and Quantitative Data by Duong, Tina, Staunton, Hannah, Braid, Jessica, Barriere, Aurelie, Trzaskoma, Ben, Gao, Ling, Willgoss, Tom, Cruz, Rosangel, Gusset, Nicole, Gorni, Ksenija, Randhawa, Sharan, Yang, Lida, Vuillerot, Carole

“…The 32-item Motor Function Measure (MFM32) is an assessment of motor function used to evaluate fine and gross motor ability in patients with neuromuscular…”
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Understanding the relationship between the 32-item motor function measure and daily activities from an individual with spinal muscular atrophy and their caregivers' perspective: a two-part study by Duong, Tina, Braid, Jessica, Staunton, Hannah, Barriere, Aurelie, Petridis, Fani, Reithinger, Johannes, Cruz, Rosangel, Jarecki, Jill, De Lemus, Mencia, Gusset, Nicole, Broekgaarden, Ria, Randhawa, Sharan, Flynn, Jessica, Arbuckle, Rob, Reif, Sonia, Yang, Lida, De Martini, Angela, Vuillerot, Carole

“…The 32-item Motor Function Measure (MFM32) is a clinician-reported outcome measure used to assess the functional abilities of individuals with neuromuscular…”
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Correction to: Understanding the relationship between the 32-item motor function measure and daily activities from an individual with spinal muscular atrophy and their caregivers’ perspective: a two-part study by Duong, Tina, Braid, Jessica, Staunton, Hannah, Barriere, Aurelie, Petridis, Fani, Reithinger, Johannes, Cruz, Rosangel, Jarecki, Jill, De Lemus, Mencia, Gusset, Nicole, Broekgaarden, Ria, Randhawa, Sharan, Flynn, Jessica, Arbuckle, Rob, Reif, Sonia, Yang, Lida, De Martini, Angela, Vuillerot, Carole

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Reverse Engineering of Digital Measures: Inviting Patients to the Conversation by Clay, Ieuan, Peerenboom, Nele, Connors, Dana E, Bourke, Steven, Keogh, Alison, Wac, Katarzyna, Gur-Arie, Tova, Baker, Justin, Bull, Christopher, Cereatti, Andrea, Cormack, Francesca, Eggenspieler, Damien, Foschini, Luca, Ganea, Raluca, Groenen, Peter MA, Gusset, Nicole, Izmailova, Elena, Kanzler, Christoph M, Leyens, Lada, Lyden, Kate, Mueller, Arne, Nam, Julian, Ng, Wan-Fai, Nobbs, David, Orfaniotou, Foteini, Perumal, Thanneer Malai, Piwko, Wojciech, Ries, Anja, Scotland, Alf, Taptiklis, Nick, Torous, John, Vereijken, Beatrix, Xu, Shuai, Baltzer, Laurenz, Vetter, Thorsten, Goldhahn, Jörg, Hoffmann, Steven C

“…Background: Digital measures offer an unparalleled opportunity to create a more holistic picture of how people who are patients behave in their real-world…”
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Importance of Patient Involvement in the Value Assessment Process: On the Way Towards Personalised Treatments by Gusset, Nicole

“…Usually, in a discussion, an individual can only take one position and wearing two hats at the same time will naturally cause conflicts of interest. [...]it is…”
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Systematic Literature Review of the Natural History of Spinal Muscular Atrophy: Motor Function, Scoliosis, and Contractures by Aponte Ribero, Valerie, Martí, Yasmina, Batson, Sarah, Mitchell, Stephen, Gorni, Ksenija, Gusset, Nicole, Oskoui, Maryam, Servais, Laurent, Sutherland, C Simone

“…Spinal muscular atrophy (SMA) is a progressive neuromuscular disorder associated with continuous motor function loss and complications, such as scoliosis and…”
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Understanding European patient expectations towards current therapeutic development in spinal muscular atrophy by Gusset, Nicole, Stalens, Caroline, Stumpe, Eva, Klouvi, Lori, Mejat, Alexandre, Ouillade, Marie-Christine, de Lemus, Mencía

“…•Most patients with SMA in Europe do not have access to the approved treatment.•Stabilization following treatment is considered progress.•Traditional SMA type…”
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Disease impact on general well-being and therapeutic expectations of european type II and type III spinal muscular atrophy patients by Rouault, Françoise, Christie-Brown, Vanessa, Broekgaarden, Ria, Gusset, Nicole, Henderson, Doug, Marczuk, Patryk, Schwersenz, Inge, Bellis, Gil, Cottet, Christian

“…Highlights • Descriptive analysis of Type II and Type III SMA from a European patients perspective • 837 replies to a survey made by SMA-Europe directly…”
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Delphi Consensus Panel on Assessing Clinically Meaningful Treatment Outcomes in Adults Living with Spinal Muscular Atrophy (SMA) (P2-11.007) by Sully, Kate, Chiriboga, Claudia, Duong, Tina, Erbas, Yasemin, Glascock, Jacqueline, Gusset, Nicole, Muni-Lofra, Robert, O’Connell, Colleen, Vázquez-Costa, Juan F., Walter, Maggie C., Guittari, Carol Jean, Cochrane, James, Townson, Louisa D., Kufakwaro, Jason, Marciniak, Anne, Riley, Danielle, Nam, Julian

“…Abstract only…”
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A Decision for Life – Treatment decisions in newly diagnosed families with spinal muscular atrophy (SMA) by Gusset, Nicole, Erbas, Yasemin, Germanenko, Olga, Rucinski, Kacper, Stumpe, Eva, de Lemus, Mencía

“…•With several treatments available, families with a child with SMA are faced with challenging decisions.•In order to make informed decisions, families need to…”
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Patient and parent oriented tools to assess health-related quality of life, activity of daily living and caregiver burden in SMA. Rome, 13 July 2019 by Mercuri, Eugenio, Messina, Sonia, Montes, Jacqueline, Muntoni, Francesco, Sansone, Valeria A., Antonaci, Laura, Civitello, Matt, Coratti, Giorgia, Lemus, Mencia de, Sanctis, Roberto de, Avexis, Marcus Droege, Duong, Tina, Finkel, Richard, Frongia, Anna Lia, Roche, Ksenija Gorni, Heatwole, Chad, Gusset, Nicole, Henricson, Erik, Mayhew, Anna, Marchesi, Chiara, Mercuri, Eugenio, Messina, Sonia, Montes, Jacqueline, Muntoni, Francesco, Pasternak, Amy, Pechmann, Astrid, Pera, Maria Carmela, Rubino, Ivana, Sansone, Valeria, Schroth, Mary, Trundell, Dylan, Straub, Volker

“…•To explore the different PROMS currently or previously used in SMA.•To define the domains explored by each tool in relation to age and SMA type.•To report the…”
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Medical-ethical recommendations: preimplantation genetic testing PGT by Swiss Medical Weekly

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254th ENMC international workshop. Formation of a European network to initiate a European data collection, along with development and sharing of treatment guidelines for adult SMA patients. Virtual meeting 28 – 30 January 2022 by Walter, Maggie C., Laforêt, Pascal, van der Pol, W. Ludo, Pegoraro, Elena, Attarian, Shahram, Bartels, Bart, Gorni, Ksenija, Goemans, Nathalie, Gusset, Nicole, Hodgkinson, Victoria, Hagenacker, Tim, Kirschner, Janbernd, Klein, Andrea, Kostera-Pruszczyk, Anna, Lochmüller, Hanns, Marini-Bettolo, Chiara, Mercuri, Eugenio, Muni-Lofra, Robert, Ouillade, Laetitia, Quinlivan, Rosaline, Papadopoulos, Constantinos, Prigent, Hélène, Salort-Campana, Emmanuelle, Sansone, Valeria A, Smit, Rivka, Smeriglio, Piera, Thiele, Simone, Tichler, Ben, Van den Bergh, Peter, Vazquez-Costa, Juan F, Vissing, John

“…•Worldwide, data collections and registries, such as TREAT-NMD patient registries, iSMAC and SMArtCARE have been implemented as important tools to get more…”
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Patientenpartizipation in der pädiatrischen Versorgungsforschung am Universitätsklinikum Freiburg: von der Projektbeteiligung zum Patientenbeirat by Langer, Thorsten, Gusset, Nicole, Pechmann, Astrid, Stumpe, Eva, Dürr, Susanne, Mund, Annette, Matilainen, Julia, Meyer, Sylvia, Barth, Michael, Haddad, Anneke

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Zeitschrift fur Evidenz, Fortbildung und Qualitat im Gesundheitswesen by Langer, Thorsten, Gusset, Nicole, Pechmann, Astrid, Stumpe, Eva, Dürr, Susanne, Mund, Annette, Matilainen, Julia, Meyer, Sylvia, Barth, Michael, Haddad, Anneke

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