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Essai en cours de recrutement = Essai en cours de recrutement; Essai en cours = Essai en cours; Financé par un membre IRDiRC = ; Membre d'un ERN =

Essai(s) clinique(s) national(aux)

ALLEMAGNE

Bayern
MÜNCHEN

ESPAGNE

Madrid
MADRID

Essai en cours de recrutementEssai en coursSetmelanotide (RM-493): Phase 2 Treatment Trial in Patients With Rare Genetic Disorders of Obesity - ES
Hospital Infantil Universitario Niño Jesús
Servicio de Endocrinología, Crecimiento y Metabolismo

FRANCE

ILE-DE-FRANCE
PARIS

PAYS-BAS

Groningen
GRONINGEN

POLOGNE

Warszawa
WARSAW

ROYAUME-UNI

Greater Manchester
ADDRESS: NOT PROVIDED - UK

ROYAUME-UNI

West Midlands
BIRMINGHAM

TCHEQUE, REPUBLIQUE

Capital City Prague
PRAHA

Essai en coursFIELD : A Study of Two Fabrazyme (agalsidase beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms (Phase IIIb) - CZ
Vseobecna fakultni nemocnice a 1. lekárska fakulta UK
General Teaching Hospital and 1st Faculty of Medicine

ALLEMAGNE

Baden-Württemberg
FREIBURG

Essai en coursFOR-DMD: Double-blind randomized phase III trial to find optimum steroid regimen for Duchenne Muscular Dystrophy - DE
Zentrum für Kinder- und Jugendmedizin Freiburg
Klinik für Neuropädiatrie und Muskelerkrankungen

ALLEMAGNE

Baden-Württemberg
HEIDELBERG

ALLEMAGNE

Baden-Württemberg
HEIDELBERG

Essai en coursAn open-label, phase 1/2 study of melflufen and dexamethasone for patients with al amyloidosis following at least one prior line of therapy - DE
Zentrum für Innere Medizin (Krehl-Klinik)
Abteilung Innere Medizin V - Hämatologie, Onkologie und Rheumatologie

ALLEMAGNE

Bayern
MÜNCHEN

ALLEMAGNE

Bayern
MÜNCHEN

CANADA

Ontario
LONDON

ETATS-UNIS

Florida
GAINESVILLE

ETATS-UNIS

New York
ROCHESTER

FRANCE

ILE-DE-FRANCE
PARIS

IRLANDE

County Dublin
DUBLIN

ITALIE

CAMPANIA
NAPOLI

Essai en cours(FOR-DMD): Duchenne Muscular Dystrophy: Double-blind Randomized Trial to Find Optimum Steroid Regimen
AOU Università degli Studi della Campania "Luigi Vanvitelli" - Centro storico
Servizio di Cardiomiologia e Genetica Medica

ITALIE

LAZIO
ROMA

Essai en cours de recrutementEssai en coursOpen Label, Long-term Safety, Tolerability, and Efficacy Study of GIVINOSTAT in All DMD Patients Who Have Been Previously Treated in One of the GIVINOSTAT Studies - IT
IRCCS Ospedale Pediatrico Bambino Gesù - SEDE SAN PAOLO
Unità di Malattie Neuromuscolari e Neurodegenerative - Laboratorio di Medicina Molecolare

ROYAUME-UNI

Cambridgeshire
CAMBRIDGE

ROYAUME-UNI

Greater London
LONDON

Essai en coursA randomized open-label multicenter phase III trial of Melphalan and Dexamethasone (MDex) versus Bortezomib, Melphalan and Dexamethasone (BMDex) for untreated patients with systemic light-chain (AL) amyloidosis - UK
Joint UCLH/UCL Biomedical Research and Development (R&D) Unit
University College London (UCL) and University College London Hospitals NHS Trust (UCLH)

ROYAUME-UNI

Greater London
LONDON

ROYAUME-UNI

Tyne & Wear
NEWCASTLE UPON TYNE

Essai en cours de recrutementEssai en coursFinancé par un membre IRDiRCA Phase III Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - UK
Newcastle upon Tyne Hospitals NHS Trust
John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine

ROYAUME-UNI

Tyne & Wear
NEWCASTLE UPON TYNE

Essai en coursAn open-label extension study of the long-term safety, tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular Dystrophy (phase III) - UK
Newcastle upon Tyne Hospitals NHS Trust
John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine

ROYAUME-UNI

Tyne & Wear
NEWCASTLE UPON TYNE

Essai en coursAn Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy (phase III) - UK
Newcastle upon Tyne Hospitals NHS Trust
John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine

SUISSE

Suisse Alémanique
BASEL

AUTRICHE

TIROL
INNSBRUCK

FRANCE

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

ROYAUME-UNI

Greater London
LONDON

Essai(s) clinique(s) multinational(aux)

ETATS-UNIS

Washington
ADDRESS: NOT PROVIDED - US

ETATS-UNIS

Washington
ADDRESS: NOT PROVIDED - US

ROYAUME-UNI

Tyne & Wear
NEWCASTLE UPON TYNE

Financé par un membre IRDiRCSCOPE-DMD: Consortium for Products across Europe in Duchenne Muscular Dystrophy
Newcastle upon Tyne Hospitals NHS Trust
John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine