5-Year watch: how does a duchenne drug perform in everyday life?
NCT ID NCT02369731
Summary
This study monitored the safety and real-world effectiveness of the drug Translarna (ataluren) for people with Duchenne Muscular Dystrophy. It followed 316 patients across Europe for at least five years as they received the drug as part of their normal medical care. The goal was to gather long-term safety data and see how well the treatment worked outside of controlled clinical trials.
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Contacts and locations
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Locations
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AO Padova Clinica Neurologica
Padua, 35128, Italy
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AOU Policlinico "G. Martino"
Messina, 98125, Italy
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ASST Grande Ospedale Metropolitano Niguarda
Milan, 20162, Italy
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Addenbrooke's Hospital
Cambridge, CB2 0QQ, United Kingdom
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Ahepa University Hospital
Thessaloniki, 54621, Greece
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Bambino Gesù Children's Hospital
Rome, 00146, Italy
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Birmingham Heartlands Hospital
Birmingham, B9 5SS, United Kingdom
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Bristol Children's Hospital
Bristol, BS2 8BJ, United Kingdom
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Bērnu klīniskā universitātes slimnīca
Riga, Latvia
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CHRU de Brest Hôpital Morvan
Brest, 29200, France
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CHRU de Lille
Lille, 59037, France
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CHRU de Tours Hôpital Bretonneau
Tours, 37044, France
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CHU de Bordeaux Hôpital Pellegrin Enfant
Bordeaux, 33076, France
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CHU de Clermont-Ferrand, hôpital Estaing
Clermont-Ferrand, 63003, France
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CHU de Marseille Hôpital de la Timone
Marseille, 13385, France
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CHU de Martinique - Hôpital P. Zobda-Quitman
Fort de France Cedex, 97261, France
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CHU de Nancy Hôpital de Brabois-Enfants Rue du Morvan
Vandœuvre-lès-Nancy, 54400, France
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CHU de Nantes
Nantes, 44093, France
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CHU de Reims American Memorial Hospital
Reims, 51092, France
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CHU de Saint-Etienne Hôpital Bellevue
Saint-Etienne, 42055, France
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CHU de Strasbourg
Strasbourg, 67098, France
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CHU de Toulouse Hôpital Pierre Paul Riquet
Toulouse, 31059, France
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CHU de la Réunion - GHSR - GH Sud-Réunion
Saint-Pierre, 97448, France
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Cardiomiologia e Genetica Medica Primo Policlinico
Naples, 80138, Italy
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Centro Hospitalar Lisboa Norte EPE - Hospital de Santa Maria
Lisbon, 1649-035, Portugal
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Centro Hospitalar do Porto EPE - Hospital Geral de Santo António
Porto, 4099-001, Portugal
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Centro Hospitalar e Universitário de coimbra EPE - Hospital Pediátrico de Coimbra
Coimbra, 3000-075, Portugal
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Children Hospital - Children's Neurosciences Leeds TEACHING HOSPITALS NHS Trust
Leeds, LS1 3EX, United Kingdom
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Children's Hospital "Aghia Sophia"
Athens, 11527, Greece
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Children's Hospital Alder Hey Children's Hospital NHS Trust
Liverpool, L12 2AP, United Kingdom
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Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, 20122, Italy
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Fondazione Policlinico Universitario Agostino Gemelli
Roma, 00168, Italy
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Göteborgs Universitet Avdelningen för pediatrik vid institutionen för kliniska vetenskaper
Gothenburg, 41685, Sweden
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Hadassah Mount Scopus Medical Center
Jerusalem, 91240, Israel
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Hospices Civils de Lyon Groupement Hospitalier Est - Bâtiment "Les Tilleuls"
Bron, 69677, France
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Hospital das Clínicas da Universidade de São Paulo
São Paulo, 05403-000, Brazil
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Hôpital Armand Trousseau
Paris, 75012, France
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Hôpital Necker - Enfants malades
Paris, 75015, France
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II Policlinico Neurofisiopatologia (Edificio 11 G)
Naples, 80131, Italy
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IRCCS Istituto Neurologico Carlo Besta
Milan, 20133, Italy
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IRCSS Istituto delle Scienze Neurologiche
Bologna, 40133, Italy
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Istituto Giannina Gaslini
Genova, 16147, Italy
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Karolinska Institutet
Stockholm, 17176, Sweden
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Klinik und Poliklinik f. Kinder- und Jugendmedizin
Hamburg, 20246, Germany
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Klinikum Bayreuth GmbH
Bayreuth, 95445, Germany
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Krankenhaus der Barmherzigen Schwestern Linz
Linz, 4010, Austria
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MVZ Kinder- und Jugendärzte Fürth GmbH
Fürth, 90762, Germany
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Motol University Hospital
Praha 5 - Motol, 15006, Czechia
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Muskelzentrum, Schwerpunkt Neuropädiatrie
Kassel, 34125, Germany
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NIHR Lancashire Clinical Research Facility Royal Preston Hospital
Preston, PR2 9HT, United Kingdom
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Ospedale Molinette SCDU Neuropsichiatria Infantile AO Città della Salute e della Scienza di Torino
Torino, 10126, Italy
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Regina Maria Policlinica Opera Center
Bucharest, Romania
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Royal Manchester Children's Hospital
Manchester, M13 9WL, United Kingdom
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SPZ Frankfurt Mitte
Frankfurt am Main, 60316, Germany
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Schneider Medical Center Tel Aviv University
Petah Tikva, 49202, Israel
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Semmelweis Egyetem II (Semmelweis University II)
Budapest, 01094, Hungary
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Sheba Medical Center The Joseph Sagol EMG, Neuroscience Center
Ramat Gan, 52621, Israel
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Sozialmedizinisches Zentrum Süd Kaiser-Franz-Josef Spital mit Gottfried von Preyer´schen Kinderspital
Vienna, 1100, Austria
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Sozialpädiatrisches Zentrum (SPZ) Charité Berlin
Berlin, 13353, Germany
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Spitalul Clinic de Copii "Dr. Victor Gomoiu" Sectia Clinica Neurologie Pediatrica
Bucharest, 22113, Romania
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St Thomas' Hospital Children Services
London, SE1 7EH, United Kingdom
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St. Olavs hospital University children's clinical hospital
Trondheim, NO-7006, Norway
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Szent László Kórház Gyermekneurológiai szakrendelés
Budapest, Hungary
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The John Walton Muscular Dystrophy Research Centre
Newcastle, NE1 3BZ, United Kingdom
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The University Hospital Brno
Brno, 62500, Czechia
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UCL Great Ormond Street Institute of Child Health
London, WC1N 1EH, United Kingdom
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Universitaetsklinikum Essen (AoR)
Essen, 45122, Germany
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Universitaetsklinikum Freiburg
Freiburg im Breisgau, 79106, Germany
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University Children's Hospital University Medical Centre Ljubljana
Ljubljana, 1000, Slovenia
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Universitätskinderklinik Gießen
Giessen, 35385, Germany
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Universitätsklinikum Erlangen
Erlangen, 91054, Germany
Conditions
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