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

Locations

  • AO Padova Clinica Neurologica

    Padua, 35128, Italy

  • AOU Policlinico "G. Martino"

    Messina, 98125, Italy

  • ASST Grande Ospedale Metropolitano Niguarda

    Milan, 20162, Italy

  • Addenbrooke's Hospital

    Cambridge, CB2 0QQ, United Kingdom

  • Ahepa University Hospital

    Thessaloniki, 54621, Greece

  • Bambino Gesù Children's Hospital

    Rome, 00146, Italy

  • Birmingham Heartlands Hospital

    Birmingham, B9 5SS, United Kingdom

  • Bristol Children's Hospital

    Bristol, BS2 8BJ, United Kingdom

  • Bērnu klīniskā universitātes slimnīca

    Riga, Latvia

  • CHRU de Brest Hôpital Morvan

    Brest, 29200, France

  • CHRU de Lille

    Lille, 59037, France

  • CHRU de Tours Hôpital Bretonneau

    Tours, 37044, France

  • CHU de Bordeaux Hôpital Pellegrin Enfant

    Bordeaux, 33076, France

  • CHU de Clermont-Ferrand, hôpital Estaing

    Clermont-Ferrand, 63003, France

  • CHU de Marseille Hôpital de la Timone

    Marseille, 13385, France

  • CHU de Martinique - Hôpital P. Zobda-Quitman

    Fort de France Cedex, 97261, France

  • CHU de Nancy Hôpital de Brabois-Enfants Rue du Morvan

    Vandœuvre-lès-Nancy, 54400, France

  • CHU de Nantes

    Nantes, 44093, France

  • CHU de Reims American Memorial Hospital

    Reims, 51092, France

  • CHU de Saint-Etienne Hôpital Bellevue

    Saint-Etienne, 42055, France

  • CHU de Strasbourg

    Strasbourg, 67098, France

  • CHU de Toulouse Hôpital Pierre Paul Riquet

    Toulouse, 31059, France

  • CHU de la Réunion - GHSR - GH Sud-Réunion

    Saint-Pierre, 97448, France

  • Cardiomiologia e Genetica Medica Primo Policlinico

    Naples, 80138, Italy

  • Centro Hospitalar Lisboa Norte EPE - Hospital de Santa Maria

    Lisbon, 1649-035, Portugal

  • Centro Hospitalar do Porto EPE - Hospital Geral de Santo António

    Porto, 4099-001, Portugal

  • Centro Hospitalar e Universitário de coimbra EPE - Hospital Pediátrico de Coimbra

    Coimbra, 3000-075, Portugal

  • Children Hospital - Children's Neurosciences Leeds TEACHING HOSPITALS NHS Trust

    Leeds, LS1 3EX, United Kingdom

  • Children's Hospital "Aghia Sophia"

    Athens, 11527, Greece

  • Children's Hospital Alder Hey Children's Hospital NHS Trust

    Liverpool, L12 2AP, United Kingdom

  • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

    Milan, 20122, Italy

  • Fondazione Policlinico Universitario Agostino Gemelli

    Roma, 00168, Italy

  • Göteborgs Universitet Avdelningen för pediatrik vid institutionen för kliniska vetenskaper

    Gothenburg, 41685, Sweden

  • Hadassah Mount Scopus Medical Center

    Jerusalem, 91240, Israel

  • Hospices Civils de Lyon Groupement Hospitalier Est - Bâtiment "Les Tilleuls"

    Bron, 69677, France

  • Hospital das Clínicas da Universidade de São Paulo

    São Paulo, 05403-000, Brazil

  • Hôpital Armand Trousseau

    Paris, 75012, France

  • Hôpital Necker - Enfants malades

    Paris, 75015, France

  • II Policlinico Neurofisiopatologia (Edificio 11 G)

    Naples, 80131, Italy

  • IRCCS Istituto Neurologico Carlo Besta

    Milan, 20133, Italy

  • IRCSS Istituto delle Scienze Neurologiche

    Bologna, 40133, Italy

  • Istituto Giannina Gaslini

    Genova, 16147, Italy

  • Karolinska Institutet

    Stockholm, 17176, Sweden

  • Klinik und Poliklinik f. Kinder- und Jugendmedizin

    Hamburg, 20246, Germany

  • Klinikum Bayreuth GmbH

    Bayreuth, 95445, Germany

  • Krankenhaus der Barmherzigen Schwestern Linz

    Linz, 4010, Austria

  • MVZ Kinder- und Jugendärzte Fürth GmbH

    Fürth, 90762, Germany

  • Motol University Hospital

    Praha 5 - Motol, 15006, Czechia

  • Muskelzentrum, Schwerpunkt Neuropädiatrie

    Kassel, 34125, Germany

  • NIHR Lancashire Clinical Research Facility Royal Preston Hospital

    Preston, PR2 9HT, United Kingdom

  • Ospedale Molinette SCDU Neuropsichiatria Infantile AO Città della Salute e della Scienza di Torino

    Torino, 10126, Italy

  • Regina Maria Policlinica Opera Center

    Bucharest, Romania

  • Royal Manchester Children's Hospital

    Manchester, M13 9WL, United Kingdom

  • SPZ Frankfurt Mitte

    Frankfurt am Main, 60316, Germany

  • Schneider Medical Center Tel Aviv University

    Petah Tikva, 49202, Israel

  • Semmelweis Egyetem II (Semmelweis University II)

    Budapest, 01094, Hungary

  • Sheba Medical Center The Joseph Sagol EMG, Neuroscience Center

    Ramat Gan, 52621, Israel

  • Sozialmedizinisches Zentrum Süd Kaiser-Franz-Josef Spital mit Gottfried von Preyer´schen Kinderspital

    Vienna, 1100, Austria

  • Sozialpädiatrisches Zentrum (SPZ) Charité Berlin

    Berlin, 13353, Germany

  • Spitalul Clinic de Copii "Dr. Victor Gomoiu" Sectia Clinica Neurologie Pediatrica

    Bucharest, 22113, Romania

  • St Thomas' Hospital Children Services

    London, SE1 7EH, United Kingdom

  • St. Olavs hospital University children's clinical hospital

    Trondheim, NO-7006, Norway

  • Szent László Kórház Gyermekneurológiai szakrendelés

    Budapest, Hungary

  • The John Walton Muscular Dystrophy Research Centre

    Newcastle, NE1 3BZ, United Kingdom

  • The University Hospital Brno

    Brno, 62500, Czechia

  • UCL Great Ormond Street Institute of Child Health

    London, WC1N 1EH, United Kingdom

  • Universitaetsklinikum Essen (AoR)

    Essen, 45122, Germany

  • Universitaetsklinikum Freiburg

    Freiburg im Breisgau, 79106, Germany

  • University Children's Hospital University Medical Centre Ljubljana

    Ljubljana, 1000, Slovenia

  • Universitätskinderklinik Gießen

    Giessen, 35385, Germany

  • Universitätsklinikum Erlangen

    Erlangen, 91054, Germany

Conditions

Explore the condition pages connected to this study.