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192 Ergebnis(se)

Nationale klinische Studie(n)

SHINE: An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443 - BE Erasme Hospital - ULB Centre de Référence Neuromusculaire Erasme-HUDERF

RAINBOWFISH: An Open-Label Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy - BE Centre Hospitalier Régional de la Citadelle Centre de Référence des Maladies Neuromusculaires (CRMN)

JEWELFISH: An Open-Label Study to Investigate the Safety, Tolerability, and Pharmacokinetics/Pharmacodynamics of RO7034067 in Adult and Pediatric Patients With Spinal Muscular Atrophy - BE Ghent University Hospital - UZ Gent NMRC UZ Gent

DEVOTE: Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - ES Institution: Information not provided - ES

A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients With Infantile-onset Spinal Muscular Atrophy - US Institution: Information not provided - US

TAMDMD (Tamoxifen in Duchenne muscular dystrophy): Multicentre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trial - BE Erasme Hospital - ULB Centre de Référence Neuromusculaire Erasme-HUDERF

SPR1NT: A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2 - BE Centre Hospitalier Régional de la Citadelle Centre de Référence des Maladies Neuromusculaires (CRMN)

An open-label safety, tolerability and pharmacokinetics study of Eteplirsen in young patients with Duchenne dystrophy amenable to exon 51 skipping - BE Ghent University Hospital - UZ Gent NMRC UZ Gent

STR1VE-EU: Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS 101 by Intravenous Infusion - BE Ghent University Hospital - UZ Gent NMRC UZ Gent

Long-term, open-label extension study for patients with Duchenne muscular dystrophy enrolled in clinical trials evaluating Casimersen or Golodirsen - BE Ghent University Hospital - UZ Gent NMRC UZ Gent

MOMENTUM: A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment - BE Ghent University Hospital - UZ Gent NMRC UZ Gent

An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen in Patients With Duchenne Muscular Dystrophy Who Have Completed Study 4658-102 - BE Ghent University Hospital - UZ Gent NMRC UZ Gent

SUNFISH: A two-part seamless, multi-center randomized, placebo-controlled, double blind study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of RO7034067 in type 2 and 3 spinal muscular atrophy patients - BE Ghent University Hospital - UZ Gent NMRC UZ Gent

FIREFISH: A Two Part Seamless, Open-label, Multicenter Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants With Type 1 Spinal Muscular Atrophy - BE Ghent University Hospital - UZ Gent NMRC UZ Gent

SIDEROS: A Phase III Double-blind, Randomized, Placebo-Controlled Study Assessing the Efficacy, Safety and Tolerability of Idebenone in Patients With Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - BE UZ Leuven - Campus Gasthuisberg Kinderen neurologie / Paediatric Neurology

SIDEROS-E: A phase III open-label extension study to assess the long-term safety and efficacy of idebenone in patients with Duchenne muscular dystrophy (DMD) who completed the SIDEROS study - BE UZ Leuven - Campus Gasthuisberg Kinderen neurologie / Paediatric Neurology

A phase I/II, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO051 in patients with Duchenne muscular dystrophy - BE UZ Leuven - Campus Gasthuisberg NMRC (afdeling kinderen) - UZ Leuven

A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of BMS-986089 in Ambulatory Boys With Duchenne Muscular Dystrophy - BE UZ Leuven - Campus Gasthuisberg NMRC (afdeling kinderen) - UZ Leuven

A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - BE UZ Leuven - Campus Gasthuisberg NMRC (afdeling kinderen) - UZ Leuven

Open 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 -BE UZ Leuven - Campus Gasthuisberg NMRC (afdeling kinderen) - UZ Leuven

Relations Between Fitness Status and the Severity of Myotonia in Patients With Congenital Myotonia Rigshospitalet Neuromuscular research unit

High Intensity Training in Patients With Spinal and Bulbar Muscular Atrophy Rigshospitalet Neuromuscular research unit

Kann eine Behandlung mit humanan Wachstumshormonen die Muskelkraft bei Patienten mit spinaler Muskelatrophie Typ II und III erhöhen (Phase-II-Studie)? Zentrum für Kinder- und Jugendmedizin Freiburg Klinik für Neuropädiatrie und Muskelerkrankungen

FOR-DMD: Doppelblinde randomisierte Phase III-Studie zur Steroid-Therapie optimierung bei Muskeldystrophie Typ Duchenne Zentrum für Kinder- und Jugendmedizin Freiburg Klinik für Neuropädiatrie und Muskelerkrankungen

Eine offene mehrteilige erste Studie am Menschen von LMI070 bei Kindern mit spinaler Muskelatrophie Typ 1 (Phase I/II) Zentrum für Kinder- und Jugendmedizin Freiburg Klinik für Neuropädiatrie und Muskelerkrankungen

Eine offene Verlängerungsstudie für Patienten mit spinaler Muskelatrophie, die zuvor an den klinischen Studien mit ISIS 396443 teilgenommen haben (Phase III) Friedrich-Baur-Institut Friedrich-Baur-Institut an der Neurologischen Klinik und Poliklinik

OLEOS: Multizentrische, offene, einarmige Studie zur Untersuchung der Langzeit Sicherheit, Verträglichkeit und Wirksamkeit von 10mg/kg Olesoxime bei Patienten mit spinaler Muskelatrophie (Phase II) Friedrich-Baur-Institut Friedrich-Baur-Institut an der Neurologischen Klinik und Poliklinik

Eine Offene Langzeitsicherheits-, Verträglichkeits- und Wirksamkeitsstudie von GIVINOSTAT bei allen DMD-Patienten, die zuvor in einer der GIVINOSTAT-Studien behandelt wurden (Phase III) Institution: Information not provided - DE

TAMDMD: Tamoxifen bei Muskeldystrophie Duchenne - Eine multizentrische, randomisierte, doppelblinde, placebo-kontrollierte Phase 3 Studie zur Untersuchung der Sicherheit und Wirksamkeit über 48 Wochen DRK Kliniken Berlin Westend Klinik für Kinder- und Jugendmedizin

Eine offene mehrteilige erste Studie am Menschen von LMI070 bei Kindern mit spinaler Muskelatrophie Typ 1 (Phase I/II) Universitätsklinikum Essen Klinik für Kinderheilkunde I - Bereich Neuropädiatrie und SPZ

TAMDMD: Tamoxifen bei Muskeldystrophie Duchenne - Eine multizentrische, randomisierte, doppelblinde, placebo-kontrollierte Phase 3 Studie zur Untersuchung der Sicherheit und Wirksamkeit über 48 Wochen Universitätsklinikum Essen Klinik für Kinderheilkunde I - Bereich Neuropädiatrie und SPZ

EPIDYS-Studie: Randomisierte, doppelblinde, placebokontrollierte, multizentrische Studie zur Beurteilung der Wirksamkeit und Sicherheit von Givinostat bei gehfähigen Patienten mit Duchenne-Muskeldystrophie Universitätsklinikum Essen Klinik für Kinderheilkunde I - Bereich Neuropädiatrie und SPZ

Eine randomisierte, doppelblinde, Placebo-kontrollierte Studie zur Beurteilung der Wirksamkeit, Sicherheit und Verträglichkeit von BMS-986089 bei ambulant behandelten Jungen mit Duchenne-Muskeldystrophie Universitätsklinikum Essen

CARNIVAL Type I - Internationale, multizentrische Phase-I/II-Therapiestudie: Sicherheit und Wirksamkeit der Valproinsäure und Carnitin bei Kindern mit Spinaler Muskelatrophie (SMA) Typ I Universitätsklinikum Köln Pädiatrische Neurologie und SPZ

A Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy-FR Institution: Information not provided - FR

A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids (SIDEROS)-IE Children's Health Ireland @ Temple Street Department of Neurology

Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy (PolarisDMD)-IE Children's Health Ireland @ Temple Street Department of Neurology

(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

A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study With Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys With Duchenne Muscular Dystrophy (DMD) - IT Institution: Information not provided - IT

A Double-Blind, Placebo-Controlled, Multi-Center Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - IT A.O.U. Sant'Anna - Polo Chimico-Bio-Medico U.O.C. di Genetica Medica

Long 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

(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

Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion Tokyo Women's Medical University

Transplantation of Myoblasts to Duchenne Muscular Dystrophy (DMD) Patients (Phase I-II) London Health Sciences Centre Children's Hospital

A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess the Effect, Safety, Tolerability and Pharmacokinetics of Multiple Subcutaneous Doses of PRO044 in Patients With Duchenne Muscular Dystrophy LUMC - Leids Universitair Medisch Centrum Polikliniek Neurologie

A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination With Systemic Corticosteroids in Subjects With Non-ambulatory Duchenne Muscular Dystrophy (DMD) - AT Institution: Information not provided - AT

SIDEROS: A Phase III Double-blind, Randomized, Placebo-Controlled Study Assessing the Efficacy, Safety and Tolerability of Idebenone in Patients With Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - AT Gottfried von Preyer'sches Kinderspital Abteilung für Kinder- und Jugendheilkunde

SIDEROS-E: A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Who Completed the SIDEROS Study - AT Gottfried von Preyer'sches Kinderspital Abteilung für Kinder- und Jugendheilkunde

A phase I/II, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO051 in patients with Duchenne muscular dystrophy - SE The Queen Silvia Children's Hospital - Sahlgrenska Department of Pediatrics

TAMDMD: Tamoxifen in Duchenne muscular dystrophy A multicentre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trial - CH Universitäts-Kinderspital beider Basel UKBB Die Abteilung für Neuro- und Entwicklungspädiatrie

SIDEROS-E: A phase III open-label extension study to assess the long-term safety and efficacy of idebenone in patients with Duchenne muscular dystrophy (DMD) who completed the SIDEROS study - CH Universitäts-Kinderspital beider Basel UKBB Die Abteilung für Neuro- und Entwicklungspädiatrie

A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of BMS-986089 in Ambulatory Boys With Duchenne Muscular Dystrophy - GB Institution: Information not provided - CH

Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy (Phase III) - ES Hospital Universitario Virgen del Rocío Unidad de Neurología Pediátrica

SIDEROS-E: A phase III open-label extension study to assess the long-term safety and efficacy of idebenone in patients with Duchenne muscular dystrophy (DMD) who completed the SIDEROS study - ES Hospital Universitario Virgen del Rocío

SIDEROS-E: A phase III open-label extension study to assess the long-term safety and efficacy of idebenone in patients with Duchenne muscular dystrophy (DMD) who completed the SIDEROS study - ES Hospital Universitari Vall d'Hebron

SIDEROS: A Phase III Double-blind, Randomized, Placebo-Controlled Study Assessing the Efficacy, Safety and Tolerability of Idebenone in Patients With Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - ES Hospital Universitari Vall d'Hebron Servicio de Neurología Pediátrica

Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy (Phase III) - ES Hospital Universitari Vall d'Hebron Servicio de Neurología Pediátrica

An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443 (Phase III) - ES Hospital Universitari Vall d'Hebron Área de Genética Clínica y Molecular

A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy (Phase III) - ES Hospital de la Santa Creu i Sant Pau Servicio de Neurología

An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443 (Phase III) - ES Hospital Sant Joan de Déu Barcelona Unidad de Enfermedades Neuromusculares

Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy (Phase III) - ES Hospital Sant Joan de Déu Barcelona Unidad de Enfermedades Neuromusculares

A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy (Phase III) - ES Hospital Sant Joan de Déu Barcelona Servicio de Neurología

TAMDMD: Tamoxifen in Duchenne muscular dystrophy A multicentre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trial - ES Hospital Sant Joan de Déu Barcelona Servicio de Neurología

SIDEROS-E: A phase III open-label extension study to assess the long-term safety and efficacy of idebenone in patients with Duchenne muscular dystrophy (DMD) who completed the SIDEROS study - ES Hospital Sant Joan de Déu Barcelona Servicio de Neurología

A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy (Phase III) - ES Hospital Universitario y Politécnico La Fe Servicio de Neurología

SIDEROS-E: A phase III open-label extension study to assess the long-term safety and efficacy of idebenone in patients with Duchenne muscular dystrophy (DMD) who completed the SIDEROS study - ES Hospital Universitario y Politécnico La Fe Servicio de Neurología

Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy (Phase III) - ES Hospital Universitario y Politécnico La Fe Servicio de Neurología

Open 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 - ES Institution: Information not provided - ES

Long-term, open-label extension study for patients with Duchenne muscular dystrophy enrolled in clinical trials evaluating casimersen or golodirsen - ES Institution: Information not provided - ES

A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination With Systemic Corticosteroids in Subjects With Non-ambulatory Duchenne Muscular Dystrophy (DMD) - ES Institution: Information not provided - ES

The TOPAZ study: Phase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients with Later-Onset Spinal Muscular Atrophy - ES Institution: Information not provided - ES

Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy (Phase III) - ES Hospital Universitario 12 de Octubre

SIDEROS-E: A phase III open-label extension study to assess the long-term safety and efficacy of idebenone in patients with Duchenne muscular dystrophy (DMD) who completed the SIDEROS study - ES Hospital Universitario La Paz

An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443 (Phase III) - ES Hospital Universitario La Paz Servicio de Neurología infantil

Optical Imaging of Dystrophic and Damaged Muscle UNIVERSITY OF FLORIDA

A Phase 2 Randomized, Double-blind, Placebo-controlled, Multiple Ascending Dose Study To Evaluate The Safety, Efficacy, Pharmacokinetics And Pharmacodynamics Of Pf-06252616 In Ambulatory Boys With Duchenne Muscular Dystrophy - US Kennedy Krieger Institute

SHINE: An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443 - BE
Erasme Hospital - ULB

Centre de Référence Neuromusculaire Erasme-HUDERF

RAINBOWFISH: An Open-Label Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy - BE
Centre Hospitalier Régional de la Citadelle

Centre de Référence des Maladies Neuromusculaires (CRMN)

JEWELFISH: An Open-Label Study to Investigate the Safety, Tolerability, and Pharmacokinetics/Pharmacodynamics of RO7034067 in Adult and Pediatric Patients With Spinal Muscular Atrophy - BE
Ghent University Hospital - UZ Gent

NMRC UZ Gent

DEVOTE: Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - ES
Institution: Information not provided - ES

A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients With Infantile-onset Spinal Muscular Atrophy - US
Institution: Information not provided - US

TAMDMD (Tamoxifen in Duchenne muscular dystrophy): Multicentre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trial - BE
Erasme Hospital - ULB

Centre de Référence Neuromusculaire Erasme-HUDERF

SPR1NT: A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2 - BE
Centre Hospitalier Régional de la Citadelle

Centre de Référence des Maladies Neuromusculaires (CRMN)

An open-label safety, tolerability and pharmacokinetics study of Eteplirsen in young patients with Duchenne dystrophy amenable to exon 51 skipping - BE
Ghent University Hospital - UZ Gent

NMRC UZ Gent

STR1VE-EU: Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS 101 by Intravenous Infusion - BE
Ghent University Hospital - UZ Gent

NMRC UZ Gent

Long-term, open-label extension study for patients with Duchenne muscular dystrophy enrolled in clinical trials evaluating Casimersen or Golodirsen - BE
Ghent University Hospital - UZ Gent

NMRC UZ Gent

MOMENTUM: A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment - BE
Ghent University Hospital - UZ Gent

NMRC UZ Gent

An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen in Patients With Duchenne Muscular Dystrophy Who Have Completed Study 4658-102 - BE
Ghent University Hospital - UZ Gent

NMRC UZ Gent

SUNFISH: A two-part seamless, multi-center randomized, placebo-controlled, double blind study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of RO7034067 in type 2 and 3 spinal muscular atrophy patients - BE
Ghent University Hospital - UZ Gent

NMRC UZ Gent

FIREFISH: A Two Part Seamless, Open-label, Multicenter Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants With Type 1 Spinal Muscular Atrophy - BE
Ghent University Hospital - UZ Gent

NMRC UZ Gent

SIDEROS: A Phase III Double-blind, Randomized, Placebo-Controlled Study Assessing the Efficacy, Safety and Tolerability of Idebenone in Patients With Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - BE
UZ Leuven - Campus Gasthuisberg

Kinderen neurologie / Paediatric Neurology

SIDEROS-E: A phase III open-label extension study to assess the long-term safety and efficacy of idebenone in patients with Duchenne muscular dystrophy (DMD) who completed the SIDEROS study - BE
UZ Leuven - Campus Gasthuisberg

Kinderen neurologie / Paediatric Neurology

A phase I/II, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO051 in patients with Duchenne muscular dystrophy - BE
UZ Leuven - Campus Gasthuisberg

NMRC (afdeling kinderen) - UZ Leuven

A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of BMS-986089 in Ambulatory Boys With Duchenne Muscular Dystrophy - BE
UZ Leuven - Campus Gasthuisberg

NMRC (afdeling kinderen) - UZ Leuven

A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - BE
UZ Leuven - Campus Gasthuisberg

NMRC (afdeling kinderen) - UZ Leuven

Open 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 -BE
UZ Leuven - Campus Gasthuisberg

NMRC (afdeling kinderen) - UZ Leuven

Relations Between Fitness Status and the Severity of Myotonia in Patients With Congenital Myotonia
Rigshospitalet

Neuromuscular research unit

High Intensity Training in Patients With Spinal and Bulbar Muscular Atrophy
Rigshospitalet

Neuromuscular research unit

Kann eine Behandlung mit humanan Wachstumshormonen die Muskelkraft bei Patienten mit spinaler Muskelatrophie Typ II und III erhöhen (Phase-II-Studie)?
Zentrum für Kinder- und Jugendmedizin Freiburg

Klinik für Neuropädiatrie und Muskelerkrankungen

FOR-DMD: Doppelblinde randomisierte Phase III-Studie zur Steroid-Therapie optimierung bei Muskeldystrophie Typ Duchenne
Zentrum für Kinder- und Jugendmedizin Freiburg

Klinik für Neuropädiatrie und Muskelerkrankungen

Eine offene mehrteilige erste Studie am Menschen von LMI070 bei Kindern mit spinaler Muskelatrophie Typ 1 (Phase I/II)
Zentrum für Kinder- und Jugendmedizin Freiburg

Klinik für Neuropädiatrie und Muskelerkrankungen

Eine offene Verlängerungsstudie für Patienten mit spinaler Muskelatrophie, die zuvor an den klinischen Studien mit ISIS 396443 teilgenommen haben (Phase III)
Friedrich-Baur-Institut

Friedrich-Baur-Institut an der Neurologischen Klinik und Poliklinik

OLEOS: Multizentrische, offene, einarmige Studie zur Untersuchung der Langzeit Sicherheit, Verträglichkeit und Wirksamkeit von 10mg/kg Olesoxime bei Patienten mit spinaler Muskelatrophie (Phase II)
Friedrich-Baur-Institut

Friedrich-Baur-Institut an der Neurologischen Klinik und Poliklinik

Eine Offene Langzeitsicherheits-, Verträglichkeits- und Wirksamkeitsstudie von GIVINOSTAT bei allen DMD-Patienten, die zuvor in einer der GIVINOSTAT-Studien behandelt wurden (Phase III)
Institution: Information not provided - DE

TAMDMD: Tamoxifen bei Muskeldystrophie Duchenne - Eine multizentrische, randomisierte, doppelblinde, placebo-kontrollierte Phase 3 Studie zur Untersuchung der Sicherheit und Wirksamkeit über 48 Wochen
DRK Kliniken Berlin Westend

Klinik für Kinder- und Jugendmedizin

Eine offene mehrteilige erste Studie am Menschen von LMI070 bei Kindern mit spinaler Muskelatrophie Typ 1 (Phase I/II)
Universitätsklinikum Essen

Klinik für Kinderheilkunde I - Bereich Neuropädiatrie und SPZ

TAMDMD: Tamoxifen bei Muskeldystrophie Duchenne - Eine multizentrische, randomisierte, doppelblinde, placebo-kontrollierte Phase 3 Studie zur Untersuchung der Sicherheit und Wirksamkeit über 48 Wochen
Universitätsklinikum Essen

Klinik für Kinderheilkunde I - Bereich Neuropädiatrie und SPZ

EPIDYS-Studie: Randomisierte, doppelblinde, placebokontrollierte, multizentrische Studie zur Beurteilung der Wirksamkeit und Sicherheit von Givinostat bei gehfähigen Patienten mit Duchenne-Muskeldystrophie
Universitätsklinikum Essen

Klinik für Kinderheilkunde I - Bereich Neuropädiatrie und SPZ

Eine randomisierte, doppelblinde, Placebo-kontrollierte Studie zur Beurteilung der Wirksamkeit, Sicherheit und Verträglichkeit von BMS-986089 bei ambulant behandelten Jungen mit Duchenne-Muskeldystrophie
Universitätsklinikum Essen

CARNIVAL Type I - Internationale, multizentrische Phase-I/II-Therapiestudie: Sicherheit und Wirksamkeit der Valproinsäure und Carnitin bei Kindern mit Spinaler Muskelatrophie (SMA) Typ I
Universitätsklinikum Köln

Pädiatrische Neurologie und SPZ

A Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy-FR
Institution: Information not provided - FR

A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids (SIDEROS)-IE
Children's Health Ireland @ Temple Street

Department of Neurology

Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy (PolarisDMD)-IE
Children's Health Ireland @ Temple Street

Department of Neurology

(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

A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study With Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys With Duchenne Muscular Dystrophy (DMD) - IT
Institution: Information not provided - IT

A Double-Blind, Placebo-Controlled, Multi-Center Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - IT
A.O.U. Sant'Anna - Polo Chimico-Bio-Medico

U.O.C. di Genetica Medica

Long 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

(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

Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion
Tokyo Women's Medical University

Transplantation of Myoblasts to Duchenne Muscular Dystrophy (DMD) Patients (Phase I-II)
London Health Sciences Centre

Children's Hospital

A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess the Effect, Safety, Tolerability and Pharmacokinetics of Multiple Subcutaneous Doses of PRO044 in Patients With Duchenne Muscular Dystrophy
LUMC - Leids Universitair Medisch Centrum

Polikliniek Neurologie

A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination With Systemic Corticosteroids in Subjects With Non-ambulatory Duchenne Muscular Dystrophy (DMD) - AT
Institution: Information not provided - AT

SIDEROS-E: A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Who Completed the SIDEROS Study - AT
Gottfried von Preyer'sches Kinderspital

Abteilung für Kinder- und Jugendheilkunde

A phase I/II, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO051 in patients with Duchenne muscular dystrophy - SE
The Queen Silvia Children's Hospital - Sahlgrenska

Department of Pediatrics

TAMDMD: Tamoxifen in Duchenne muscular dystrophy A multicentre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trial - CH
Universitäts-Kinderspital beider Basel UKBB

Die Abteilung für Neuro- und Entwicklungspädiatrie

SIDEROS-E: A phase III open-label extension study to assess the long-term safety and efficacy of idebenone in patients with Duchenne muscular dystrophy (DMD) who completed the SIDEROS study - CH
Universitäts-Kinderspital beider Basel UKBB

Die Abteilung für Neuro- und Entwicklungspädiatrie

A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of BMS-986089 in Ambulatory Boys With Duchenne Muscular Dystrophy - GB
Institution: Information not provided - CH

Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy (Phase III) - ES
Hospital Universitario Virgen del Rocío

Unidad de Neurología Pediátrica

SIDEROS-E: A phase III open-label extension study to assess the long-term safety and efficacy of idebenone in patients with Duchenne muscular dystrophy (DMD) who completed the SIDEROS study - ES
Hospital Universitario Virgen del Rocío

SIDEROS-E: A phase III open-label extension study to assess the long-term safety and efficacy of idebenone in patients with Duchenne muscular dystrophy (DMD) who completed the SIDEROS study - ES
Hospital Universitari Vall d'Hebron

SIDEROS: A Phase III Double-blind, Randomized, Placebo-Controlled Study Assessing the Efficacy, Safety and Tolerability of Idebenone in Patients With Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - ES
Hospital Universitari Vall d'Hebron

Servicio de Neurología Pediátrica

Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy (Phase III) - ES
Hospital Universitari Vall d'Hebron

Servicio de Neurología Pediátrica

An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443 (Phase III) - ES
Hospital Universitari Vall d'Hebron

Área de Genética Clínica y Molecular

A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy (Phase III) - ES
Hospital de la Santa Creu i Sant Pau

Servicio de Neurología

An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443 (Phase III) - ES
Hospital Sant Joan de Déu Barcelona

Unidad de Enfermedades Neuromusculares

Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy (Phase III) - ES
Hospital Sant Joan de Déu Barcelona

Unidad de Enfermedades Neuromusculares

A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy (Phase III) - ES
Hospital Sant Joan de Déu Barcelona

Servicio de Neurología

TAMDMD: Tamoxifen in Duchenne muscular dystrophy A multicentre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trial - ES
Hospital Sant Joan de Déu Barcelona

Servicio de Neurología

SIDEROS-E: A phase III open-label extension study to assess the long-term safety and efficacy of idebenone in patients with Duchenne muscular dystrophy (DMD) who completed the SIDEROS study - ES
Hospital Sant Joan de Déu Barcelona

Servicio de Neurología

A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy (Phase III) - ES
Hospital Universitario y Politécnico La Fe

Servicio de Neurología

SIDEROS-E: A phase III open-label extension study to assess the long-term safety and efficacy of idebenone in patients with Duchenne muscular dystrophy (DMD) who completed the SIDEROS study - ES
Hospital Universitario y Politécnico La Fe

Servicio de Neurología

Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy (Phase III) - ES
Hospital Universitario y Politécnico La Fe

Servicio de Neurología

Open 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 - ES
Institution: Information not provided - ES

Long-term, open-label extension study for patients with Duchenne muscular dystrophy enrolled in clinical trials evaluating casimersen or golodirsen - ES
Institution: Information not provided - ES

A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination With Systemic Corticosteroids in Subjects With Non-ambulatory Duchenne Muscular Dystrophy (DMD) - ES
Institution: Information not provided - ES

The TOPAZ study: Phase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients with Later-Onset Spinal Muscular Atrophy - ES
Institution: Information not provided - ES

Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy (Phase III) - ES
Hospital Universitario 12 de Octubre

SIDEROS-E: A phase III open-label extension study to assess the long-term safety and efficacy of idebenone in patients with Duchenne muscular dystrophy (DMD) who completed the SIDEROS study - ES
Hospital Universitario La Paz

An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443 (Phase III) - ES
Hospital Universitario La Paz

Servicio de Neurología infantil

Optical Imaging of Dystrophic and Damaged Muscle
UNIVERSITY OF FLORIDA

A Phase 2 Randomized, Double-blind, Placebo-controlled, Multiple Ascending Dose Study To Evaluate The Safety, Efficacy, Pharmacokinetics And Pharmacodynamics Of Pf-06252616 In Ambulatory Boys With Duchenne Muscular Dystrophy - US
Kennedy Krieger Institute

A Multicenter, Open-label Extension Study To Evaluate The Long Term Safety Of Pf-06252616 In Boys With Duchenne Muscular Dystrophy (Phase II) - US
Kennedy Krieger Institute

A Double-Blind, Placebo-Controlled, Multi-Center Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - IT
Sarepta Therapeutics, Inc.

An Open-label, Safety Study for Previously Treated Ataluren (PTC124) Patients With Nonsense Mutation Dystrophinopathy (Phase III) - US
PTC Therapeutics Inc.

FOR-DMD: Duchenne Muscular Dystrophy: Double-blind Randomized Trial to Find Optimum Steroid Regimen (Phase III) -US
University of Rochester

Neurology, University of Rochester

Phase I Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering the Survival Motor Neuron Gene by Self-Complementary AAV9
Nationwide Children's Hospital

Center for Gene Therapy