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Nationale klinische studie(s)
BELGIË
OOST-VLAANDEREN
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
DUITSLAND
Baden-Württemberg
FREIBURG
Can 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
DUITSLAND
Baden-Württemberg
FREIBURG
NURTURE: An Open-Label Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Subjects With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy (Phase II) - DE
Zentrum für Kinder- und Jugendmedizin Freiburg
Klinik für Neuropädiatrie und Muskelerkrankungen
DUITSLAND
Bayern
MÜNCHEN
An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443 (Phase III) - DE
Friedrich-Baur-Institut
Friedrich-Baur-Institut an der Neurologischen Klinik und Poliklinik
DUITSLAND
Bayern
MÜNCHEN
OLEOS: Multicenter, Open-Label, Single Arm Study to Evaluate Long-term Safety, Tolerability, and Effectiveness of 10 mg/kg Olesoxime in Patients with SMA (Phase II)
Friedrich-Baur-Institut
Friedrich-Baur-Institut an der Neurologischen Klinik und Poliklinik
NEDERLAND
Utrecht
UTRECHT
SPACE trial: Een Fase II, Mono-centrische, Placebogecontroleerde Cross-over Trial naar Effect en Doeltreffendheid van Pyridostigmine in Volwassen Nederlandse Patienten met Spinale Spieratrofie Type 2, 3 en 4
UMC Utrecht - Universitair Medisch Centrum Utrecht
Polikliniek Neuromusculaire Ziekten
SPANJE
Andalucía
SEVILLA
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 (Phase II) - ES
Hospital Universitario Virgen del Rocío
Unidad de Neurología Pediátrica
SPANJE
Castilla - La Mancha
ALCÁZAR DE SAN JUAN
A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101 - ES
Hospital General La Mancha Centro
SPANJE
Cataluña
BARCELONA
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
SPANJE
Cataluña
BARCELONA
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 (Phase II) - ES
Hospital Universitari Vall d'Hebron
Área de Genética Clínica y Molecular
SPANJE
Cataluña
ESPLUGUES DE LLOBREGAT
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
SPANJE
Cataluña
ESPLUGUES DE LLOBREGAT
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 (Phase II) - ES
Hospital Sant Joan de Déu Barcelona
Servicio de Neurología
SPANJE
Madrid
ADDRESS: 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
SPANJE
Madrid
MADRID
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
SPANJE
Madrid
MADRID
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 (Phase II) - ES
Hospital Universitario La Paz
Servicio de Neurología infantil
BELGIË
ARRONDISSEMENT BRUSSELS-CAPITAL
BRUSSELS
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
BELGIË
LIEGE
LIEGE
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)
BELGIË
OOST-VLAANDEREN
GENT
JEWELFISH: Een open-label onderzoek naar de veiligheid, verdraagbaarheid en farmacokinetiek/farmacodynamiek van RO7034067 bij volwassen en pediatrische patiënten met spinale spieratrofie - BE
Ghent University Hospital - UZ Gent
NMRC UZ Gent
FRANKRIJK
ILE-DE-FRANCE
PARIS
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 - FR
CHU Paris Est - Hôpital d'Enfants Armand-Trousseau
Institut I-Motion - Centre de recherche pédiatrique en pathologies neuromusculaires
SPANJE
Madrid
ADDRESS: NOT PROVIDED - ES
DEVOTE: Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - ES
Institution: Information not provided - ES
USA
Washington
ADDRESS: NOT PROVIDED - US
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
FRANKRIJK
ILE-DE-FRANCE
GARCHES
Long-Term Effect of LIAM (Lung Insufflation Assist Maneuver) on Respiratory Performance in Non-invasive Ventilated Patients Suffering From Neuromuscular Disease
CHU Paris IdF Ouest - Hôpital Raymond Poincaré
Centre d'Investigation Clinique et Technologique 805 - CIC-IT de Garches
FRANKRIJK
ILE-DE-FRANCE
GARCHES
Establishment of a Non-invasive and Indirect Measure of Volitional Pimax (PI MAX STIM)
CHU Paris IdF Ouest - Hôpital Raymond Poincaré
Service de Physiologie-Explorations Fonctionnelles
Multinationale klinische studie(s)
ITALIË
LAZIO
ROMA
Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients - coordination
IRCCS Ospedale Pediatrico Bambino Gesù - SEDE GIANICOLO
Ospedale Pediatrico Bambino Gesù
ZWITSERLAND
Suisse Alémanique
BASEL