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Résultats de la recherche - 4 results
A retrospective, non-interventional cohort study to compare adults with SMA receiving risdiplamwith untreated patients: Study design.
Walter, M.; Marlène Jagut; Haberlová, J.; Hodgkinson, V.; Yiu, E.; Murphy, L.; TREAT-NMD Global Data systems Oversight Committee (TGDOC); TREAT-NMD Global Registry Network for SMA; Roe, T.; ...
International collaboration to improve knowledge on myotonic dystrophy type 2
Chiara; Walker, Helen; Voháňka, Stanislav; Cumming, Kleed; Łusakowska, Anna; Hodgkinson, Victoria; Marjan Cosyns; Rodrigues, Miriam; Yiu, Eppie; Mazanec, Radim; Stevenson, Tanya; Kostera-Pruszczyk, Anna; ...
Collaborative data collection by TREAT-NMD Registries to support post-marketing surveillance in Spinal Muscular Atrophy
Publication Type: Scientific poster, presentation or proceeding Authors: Miriam Rodrigues; JoAnne Bullivant; Victoria Hodgkinson; TREAT-NMD Registry Curators; Marlène Jagut; Volker Straub; Nathalie ...
Affiliation to the Treat-NMD Consortium Using an Agreed Minimum Dataset Allows Small Registries and Large Registries to Collaborate Together
Publication Type: Scientific poster, presentation or proceeding Authors: Richard Roxburgh; Miriam Rodrigues; Victoria Hodgkinson; Lawrence Korngut; Benedikt Schoser; Federica Montagnese; Stephan ...