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Ensaio em fase de recrutamento = Ensaio em fase de recrutamento; Ensaio a decorrer = Ensaio a decorrer; Financiado por um membro do IRDiRC =

Ensaio (s) clínico (s) nacional (is)

FRANCA

ILE-DE-FRANCE
PARIS

ALEMANHA

Baden-Württemberg
FREIBURG

Ensaio a decorrerCan treatment with human growth hormone increase strength in spinal muscular atrophy type II and III (phase II study)?
Zentrum für Kinder- und Jugendmedizin Freiburg
Klinik für Neuropädiatrie und Muskelerkrankungen

ALEMANHA

Baden-Württemberg
FREIBURG

Ensaio a decorrerFOR-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

ALEMANHA

Baden-Württemberg
FREIBURG

Ensaio a decorrerFinanciado por um membro do IRDiRCAn Open-Label Study for Previously Treated Ataluren (PTC124) Patients With Nonsense Mutation Dystrophinopathy (Phase III) - DE
Zentrum für Kinder- und Jugendmedizin Freiburg
Klinik für Neuropädiatrie und Muskelerkrankungen

ALEMANHA

Baden-Württemberg
FREIBURG

Ensaio em fase de recrutamentoEnsaio a decorrerFinanciado por um membro do IRDiRCA Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy - DE
Zentrum für Kinder- und Jugendmedizin Freiburg
Klinik für Neuropädiatrie und Muskelerkrankungen

ALEMANHA

Baden-Württemberg
FREIBURG

ALEMANHA

Baden-Württemberg
FREIBURG

Ensaio a decorrerAn Open Label Multi-part First-in-human Study of Oral LMI070 in Infants With Type 1 Spinal Muscular Atrophy (Phase I/II) - DE
Zentrum für Kinder- und Jugendmedizin Freiburg
Klinik für Neuropädiatrie und Muskelerkrankungen

ALEMANHA

Baden-Württemberg
FREIBURG

Ensaio em fase de recrutamentoEnsaio a decorrerAn open-label safety, tolerability and pharmacokinetics study of Eteplirsen in young patients with Duchenne dystrophy amenable to exon 51 skipping - DE
Zentrum für Kinder- und Jugendmedizin Freiburg
Klinik für Neuropädiatrie und Muskelerkrankungen

ALEMANHA

Bayern
MÜNCHEN

ALEMANHA

Bayern
MÜNCHEN

ALEMANHA

Nordrhein-Westfalen
ESSEN

Ensaio a decorrerAn Open Label Multi-part First-in-human Study of Oral LMI070 in Infants With Type 1 Spinal Muscular Atrophy (Phase I/II) - DE
Universitätsklinikum Essen
Klinik für Kinderheilkunde I - Bereich Neuropädiatrie und SPZ

ALEMANHA

Nordrhein-Westfalen
ESSEN

CANADA

Ontario
LONDON

DINAMARCA

Hovedstaden
COPENHAGEN

DINAMARCA

Hovedstaden
COPENHAGEN

ESPANHA

Cataluña
ESPLUGUES DE LLOBREGAT

ESTADOS UNIDOS

Florida
GAINESVILLE

ESTADOS UNIDOS

New York
ROCHESTER

FRANCA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCA

ILE-DE-FRANCE
PARIS

IRLANDA

County Dublin
DUBLIN

ITALIA

CAMPANIA
NAPOLI

Ensaio a decorrer(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

ITALIA

LAZIO
ROMA

Ensaio em fase de recrutamentoEnsaio a decorrerOpen 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
IRCCS Ospedale Pediatrico Bambino Gesù - SEDE SAN PAOLO
Unità di Malattie Neuromuscolari e Neurodegenerative - Laboratorio di Medicina Molecolare

ITALIA

LOMBARDIA
MILANO

Ensaio em fase de recrutamentoEnsaio a decorrerLong Term Safety Study of Amifampridine Phosphate in Ambulatory Patients With Spinal Muscular Atrophy (SMA) Type 3
Fondazione IRCCS Istituto Neurologico "Carlo Besta"
U.O. Neurologia IV - Malattie Neuromuscolari e Neuroimmunologia

ITALIA

VENETO
PADOVA

Ensaio a decorrer(FOR-DMD): Duchenne Muscular Dystrophy: Double-blind Randomized Trial to Find Optimum Steroid Regimen
Azienda Ospedaliera Universitaria di Padova - Campus Biomedico Pietro d'Abano
Neuromuscular Laboratory

REINO UNIDO

Cambridgeshire
CAMBRIDGE

REINO UNIDO

Greater London
LONDON

REINO UNIDO

Greater London
LONDON

REINO UNIDO

Tyne & Wear
NEWCASTLE UPON TYNE

Ensaio em fase de recrutamentoEnsaio a decorrerFinanciado por um membro do 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

REINO UNIDO

Tyne & Wear
NEWCASTLE UPON TYNE

Ensaio a decorrerAn 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

REINO UNIDO

Tyne & Wear
NEWCASTLE UPON TYNE

Ensaio a decorrerAn 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

REINO UNIDO

Tyne & Wear
NEWCASTLE UPON TYNE

SUICA

Suisse Alémanique
BASEL

DINAMARCA

Hovedstaden
COPENHAGEN

FRANCA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

REINO UNIDO

Tyne & Wear
NEWCASTLE UPON TYNE

Ensaio a decorrerPTC124-GD-007-DMD: A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy - UK
Newcastle upon Tyne Hospitals NHS Trust
John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine

AUSTRIA

SALZBURG
SALZBURG

Ensaio a decorrerMitoFibrate CT1: Clinical study for the assessment of safety and efficacy of Bezafibrate as a drug therapy for patients with mitochondrial myopathies (phase II) - AT
Universitätsklinikum der Paracelsus Medizinischen Privatuniversität
Universitätsklinik für Kinder- und Jugendheilkunde

REINO UNIDO

Greater London
LONDON

Ensaio a decorrerTEETPIM:Trial of Erythrocyte Encapsulated Thymidine Phosphorylase In Mitochondrial Neurogastrointestinal Encephalomyopathy-GB
St George's University of London
Molecular and Clinical Sciences Reseach Institute

REINO UNIDO

Greater Manchester
ADDRESS: NOT PROVIDED - UK

REINO UNIDO

Greater London
LONDON

Ensaio (s) clínico (s) multinacional (ais)

ESTADOS UNIDOS

Washington
ADDRESS: NOT PROVIDED - US

REINO UNIDO

Tyne & Wear
NEWCASTLE UPON TYNE

Ensaio a decorrerFinanciado por um membro do 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