Vue transversale de la maladie
Myopathies
Un même point d’entrée pour les études cliniques, les découvertes scientifiques, les publications et les pistes exploratoires présentes dans Biomedical Watch.
Périmètre
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1012 résultats correspondants
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dans le périmètre Myopathies
Niveau clinique
Études et traitements 421
Interventions, programmes et essais cliniques rattachés au périmètre sélectionné.
| Molécule / élément | Maladie / population | Objectif | Phase / stade | Statut / preuve | Mécanisme / résumé | Source | Détail |
|---|---|---|---|---|---|---|---|
| SGT-003 NCT06138639 · A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE) | Myopathies | Thérapie génique | Phase 1/2 | RECRUITING | This is a Phase 3, double-blind, placebo-controlled study with the primary… À vérifier | ClinicalTrials.gov 2026-08-16 19:04:32 | Détail |
| ASP2957 NCT07052929 · Study of ASP2957 in Male Participants With X-linked Myotubular Myopathy Who Need… | Myopathies | Traitement symptomatique · Thérapie génique | Phase 1/2 | RECRUITING | X-linked myotubular myopathy (XLMTM) is a rare and serious condition prese… À vérifier | ClinicalTrials.gov 2026-08-16 19:04:31 | Détail |
| CT103A NCT07752264 · A Phase I/II Study of Eque-cel in Subjects Withimmune-mediated Necrotizing Myopa… | Myopathies | Thérapie cellulaire | Phase 1/2 | NOT_YET_RECRUITING | CAR-T autologue anti-BCMA (CT103A / equecabtagene autoleucel) visant les c… À vérifier | ClinicalTrials.gov NCT04561557 ; Cell 2025 (Qin et al.… 2026-08-16 19:04:31 | Détail |
| NKX019 NCT06733935 · A Phase 1/2 Study of NKX019 in Subjects With Immune-Mediated Diseases (Ntrust-2) | Myopathies | À vérifier | Phase 1/2 | RECRUITING | Primary objective: Safety and tolerability of NKX019, administered after l… À vérifier | ClinicalTrials.gov 2026-08-16 19:04:31 | Détail |
| RGX-202 NCT05693142 · AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular D… | Myopathies | Thérapie génique | Phase 2/3 | ACTIVE_NOT_RECRUITING | RGX-202 is a gene therapy designed to deliver a transgene for a novel micr… À vérifier | ClinicalTrials.gov 2026-08-16 18:10:04 | Détail |
| GENOMIT — Global Registry and Natural History Study for Mitochondrial… NCT05554835 · NCT05554835 | Myopathies | Remyélinisation · Remyélinisation indirecte / réparation | À vérifier | RECRUITING | The main goal of the project is provision of a global registry for mitocho… À vérifier | ClinicalTrials.gov 2026-08-16 18:10:03 | Détail |
| AIM — Autoimmune Intervention Mastery Course Study NCT05057676 · NCT05057676 | Myopathies | Traitement symptomatique | Non applicable | ACTIVE_NOT_RECRUITING | The goal of this project is to critically evaluate the effectiveness of us… À vérifier | ClinicalTrials.gov 2026-08-16 18:10:02 | Détail |
| DYNE-251 NCT05524883 · DELIVER — Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic S… | Myopathies | À vérifier | Phase 1/2 | ACTIVE_NOT_RECRUITING | The primary purpose of this study is to evaluate the safety, tolerability,… À vérifier | ClinicalTrials.gov 2026-08-16 12:59:24 | Détail |
| ENTR-601-44 NCT07037862 · ELEVATE-44 — A Study in Participants With Duchenne Muscular Dystrophy Amenable t… | Myopathies | Thérapie génique | Phase 1/2 | RECRUITING | This is a study of the investigational medicine ENTR-601-44 in participant… À vérifier | ClinicalTrials.gov 2026-08-16 12:59:24 | Détail |
| ENTR-601-45 NCT07038824 · ELEVATE-45 — A Study in Participants With Duchenne Muscular Dystrophy Amenable t… | Myopathies | Thérapie génique | Phase 1/2 | RECRUITING | This is a study of the investigational medicine ENTR-601-45 in participant… À vérifier | ClinicalTrials.gov 2026-08-16 12:59:24 | Détail |
| Scapular Stabilization Exercises Combined With Thoracic Mobilization… NCT07766148 · NCT07766148 | Myopathies | Traitement symptomatique | Non applicable | COMPLETED | Upper-back pain is common among breastfeeding mothers and may be associate… À vérifier | ClinicalTrials.gov 2026-08-16 12:59:24 | Détail |
| SGT-001 NCT03368742 · IGNITE DMD — Microdystrophin Gene Transfer Study in Adolescents and Children Wit… | Myopathies | À vérifier | Phase 1/2 | ACTIVE_NOT_RECRUITING | This is a controlled, open-label, single-ascending dose study to evaluate… À vérifier | ClinicalTrials.gov 2026-08-16 12:59:24 | Détail |
| SRP-1001 for Injection NCT06131983 · Study of SRP-1001 in Adult and Adolescent Participants With Facioscapulohumeral… | Myopathies | À vérifier | Phase 1/2 | RECRUITING | The purpose of this study is to evaluate the safety, tolerability, pharmac… À vérifier | ClinicalTrials.gov 2026-08-16 12:59:24 | Détail |
| SRP-1003 IV Infusion NCT06138743 · Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy | Myopathies | À vérifier | Phase 1/2 | RECRUITING | This is a phase 1/2a double-blinded, placebo-controlled, dose-escalating s… À vérifier | ClinicalTrials.gov 2026-08-16 12:59:24 | Détail |
| Comparison of Eccentric Muscle Training and Proprioceptive Neuromuscu… NCT06407479 · NCT06407479 | Myopathies | Ralentissement de la progression · Traitement symptomatique | Non applicable | COMPLETED | This trial compared eccentric exercise training and the proprioceptive neu… À vérifier | ClinicalTrials.gov 2026-08-16 12:59:23 | Détail |
| Descartes-08 NCT07391605 · Descartes-08 in Autoantibody Myositis | Myopathies | À vérifier | Phase 2 | RECRUITING | This is a Phase IIb study to evaluate the safety and preliminary efficacy… À vérifier | ClinicalTrials.gov 2026-08-16 12:59:23 | Détail |
| FIBOBS — Fibromyalgia Syndrome in France: The Role of Assessment and… NCT04775368 · NCT04775368 | Myopathies | Traitement symptomatique | Non applicable | COMPLETED | The FIBOBS study is a multi-centered, observational, prospective study car… À vérifier | ClinicalTrials.gov 2026-08-16 12:59:23 | Détail |
| Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC) NCT06579352 · Study of UC-MSC in DMD Patients | Myopathies | Immunomodulation · Thérapie cellulaire | Phase 2 | ACTIVE_NOT_RECRUITING | The primary objective of this study is to provide UC-MSC treatment to pati… À vérifier | ClinicalTrials.gov 2026-08-16 12:59:23 | Détail |
| ISADER — Adduction and Infraspinatus Activity in Shoulder Pain NCT07765056 · NCT07765056 | Myopathies | Traitement symptomatique · Thérapie génique | Non applicable | COMPLETED | The goal of this randomized crossover trial is to determine whether differ… À vérifier | ClinicalTrials.gov 2026-08-16 12:59:23 | Détail |
| Prednisolone NCT05412394 · Once Weekly Infant Corticosteroid Trial for DMD | Myopathies | Immunomodulation · Thérapie génique | Phase 4 | RECRUITING | The goal of this clinical trial is to learn how different treatments affec… À vérifier | ClinicalTrials.gov 2026-08-16 12:59:23 | Détail |
Découverte scientifique
Recherches et trouvailles 0
Projets précliniques, publications émergentes et programmes de recherche suivis dans le pipeline.
| Molécule / élément | Maladie / population | Objectif | Phase / stade | Statut / preuve | Mécanisme / résumé | Source | Détail |
|---|---|---|---|---|---|---|---|
| Aucune trouvaille ne correspond aux filtres. | |||||||
Bibliographie
Publications 585
Références bibliographiques de la base locale et accès à leur source originale.
| Molécule / élément | Maladie / population | Objectif | Phase / stade | Statut / preuve | Mécanisme / résumé | Source | Détail |
|---|---|---|---|---|---|---|---|
| Identifying the Diagnostic Challenges and Indicators of Orthostatic T… Data Collection of Standard Care of Patients in the EMG Section · PMID 40265716 | Myopathies | Traitement symptomatique · Thérapie génique | Publication | PubMed | Voir la publication source The diagnosis of orthostatic tremor (OT) is challenging because of its non-specific symptoms and a broad range of diffe… | Movement disorders clinical practice 2026-08-13 15:03:20 | Source |
| Utilization of Preoperative Electrodiagnostic Studies for Carpal Tunn… Data Collection of Standard Care of Patients in the EMG Section · PMID 27068003 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Given the lack of a reference standard diagnostic tool for carpal tunnel syndrome (CTS), we conducted a population-leve… | The Journal of hand surgery 2026-08-13 15:03:20 | Source |
| Applicability of an Immersive Virtual Reality Exercise Training Syste… Dual-task cycling · PMID 35878115 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Virtual reality is a computer-generated simulation of a real or imaginary three-dimensional environment that has entere… | Sports (Basel, Switzerland) 2026-08-13 15:03:19 | Source |
| Event-related brain potentials reveal enhancing and compensatory mech… Dual-task cycling · PMID 37845733 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Various neurocognitive tests have shown that cycling enhances cognitive performance compared to resting. Event-related… | BMC sports science, medicine & rehabilitation 2026-08-13 15:03:19 | Source |
| Advancing treatment of spinal muscular atrophy through inhibition of… Apitegromab · PMID 41566720 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source In spinal muscular atrophy (SMA), irreversible loss of spinal motor neurons and progressive skeletal muscle atrophy cau… | Expert review of neurotherapeutics 2026-08-13 15:03:18 | Source |
| Apitegromab for lean mass preservation during tirzepatide-induced wei… Apitegromab · PMID 42260100 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Loss of lean mass in proportion to total weight loss is observed with incretin mimetic therapies such as tirzepatide an… | Nature medicine 2026-08-13 15:03:18 | Source |
| Impact of Virtual Reality-Based Task-Oriented Training for Enhancing… Standard rehabilitation · PMID 42581516 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source BACKGROUND Stroke frequently causes persistent motor impairment and dependence in activities of daily living (ADL). Vir… | Medical science monitor : international medical journa… 2026-08-13 15:03:17 | Source |
| The Role of Radial Shockwave Therapy in Reducing Spasticity and Impro… Standard rehabilitation · PMID 42589983 | Myopathies | Traitement symptomatique | Publication | PubMed | Voir la publication source : Spasticity resulting from a stroke usually develops within a few weeks or months of the incident and occurs in approx… | Journal of clinical medicine 2026-08-13 15:03:17 | Source |
| Exploring somatosensory temporal discrimination deficits in rheumatoi… Constant Current Stimulator · PMID 41316482 | Myopathies | Ralentissement de la progression | Publication | PubMed | Voir la publication source Objective- Somatosensory deficits in rheumatoid arthritis (RA) have not been adequately investigated, despite their pot… | Advances in rheumatology (London, England) 2026-08-13 15:03:15 | Source |
| Optimizing Movement Performance with Altered Sensation: An Examinatio… Constant Current Stimulator · PMID 37759903 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Two experiments were conducted to assess the impact of induced paresthesia on movement parameters of goal-directed aimi… | Brain sciences 2026-08-13 15:03:15 | Source |
| Degradation-Robust Hue Prior Network for Low-Light Rainy Image Restor… SRP-1003 SC Injection · PMID 42590629 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Restoring images captured in low-light rainy scenes is challenging because brightness degradation and rain corruption a… | Sensors (Basel, Switzerland) 2026-08-13 15:03:14 | Source |
| Elegans-Inspired Magnetic Polyurethane Soft Robot (MPSR) for Closed V… SRP-1003 SC Injection · PMID 42590852 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Secondary hemorrhage is inevitable during the transportation of casualties in harsh environments, and reoperative treat… | Advanced healthcare materials 2026-08-13 15:03:14 | Source |
| Remifentanil attenuates LPS-induced hepatic injury by modulating NRF2… SRP-1003 IV Infusion · PMID 42584673 | Myopathies | Neuroprotection · Immunomodulation · Thérapie génique | Publication | PubMed | Voir la publication source Lipopolysaccharide (LPS)-induced sepsis causes widespread inflammation and liver dysfunction, driven by oxidative stres… | Naunyn-Schmiedeberg's archives of pharmacology 2026-08-13 15:03:13 | Source |
| Transient Neonatal Myasthenia Gravis in a Newborn Infant Presenting t… SRP-1003 IV Infusion · PMID 42583073 | Myopathies | Thérapie génique | Publication | PubMed | Voir la publication source A four-day-old female born full term via uncomplicated vaginal delivery presented to the emergency department with poor… | Cureus 2026-08-13 15:03:13 | Source |
| AOC 1044 induces exon 44 skipping and restores dystrophin protein in… AOC 1044 · PMID 40183632 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Duchenne muscular dystrophy (DMD) is a severe disorder caused by mutations in the dystrophin gene, resulting in loss of… | Nucleic acids research 2026-08-13 15:03:12 | Source |
| Antibodies to watch in 2026. delpacibart zotadirsen · PMID 41560619 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source The Antibodies to Watch article series provides annual updates on commercial late-stage clinical development, regulator… | mAbs 2026-08-13 15:03:10 | Source |
| A hospital based epidemiological study of genetically determined musc… 3 Year Follow up on ANO5 Patients · PMID 32146000 | Myopathies | Thérapie génique | Publication | PubMed | Voir la publication source We determined the prevalence of genetically determined neuromuscular diseases in adult Norwegian patients from Hordalan… | Neuromuscular disorders : NMD 2026-08-13 15:03:09 | Source |
| Clinical and genetic features of anoctaminopathy in Saudi Arabia. 3 Year Follow up on ANO5 Patients · PMID 25864073 | Myopathies | Ralentissement de la progression · Traitement symptomatique | Publication | PubMed | Voir la publication source Characterization of the phenotypic, pathological, radiological, and genetic findings in 2 Saudi Arabian families with a… | Neurosciences (Riyadh, Saudi Arabia) 2026-08-13 15:03:09 | Source |
| Individualized 3D-Printed Titanium Mesh-Assisted Alveolar Reconstruct… 3d printed Stabilization splint · PMID 42551905 | Myopathies | Remyélinisation indirecte / réparation | Publication | PubMed | Voir la publication source Medication-related osteonecrosis of the jaw is a severe complication of antiresorptive therapy that often necessitates… | The Journal of oral implantology 2026-08-13 15:03:08 | Source |
| Trueness and scanning time of complete-arch intraoral scans of edentu… 3d printed Stabilization splint · PMID 42521277 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source This in vitro study evaluated the influence of the presence of saliva in varying levels on the trueness and scanning ti… | Journal of prosthodontics : official journal of the Am… 2026-08-13 15:03:08 | Source |
Exploration — prudence
Pistes exploratoires 6
Rapprochements mécanistiques et hypothèses de recherche : ils ne constituent pas une preuve d’efficacité ni une recommandation thérapeutique.
Hypothèses non validéesÀ confirmer par des travaux précliniques, cliniques et une revue scientifique indépendante.
| Molécule / élément | Maladie / population | Objectif | Phase / stade | Statut / preuve | Mécanisme / résumé | Source | Détail |
|---|---|---|---|---|---|---|---|
| N-PPG N-PPG — modulation métabolique cérébrale | Myopathies | Remyélinisation indirecte / réparation · Neuroprotection | Hypothèse exploratoire | Rapprochement automatique non validé | Modulation métabolique et mitohormèse N-PPG est étudié dans Maladie de Huntington et documente plusieurs axes biologiques pouvant être pertinents pour Myopat… | Rapprochement Biomedical Watch · Maladie de Huntington | Détail |
| Vecteur Ple389 (ADORA2A) MiniPromoter Ple389 — ciblage striatal | Myopathies | Thérapie génique | Hypothèse exploratoire | Rapprochement automatique non validé | Expression génique ciblée dans les neurones striataux Vecteur Ple389 (ADORA2A) est étudié dans Maladie de Huntington et documente plusieurs axes biologiques pouvant être per… | Rapprochement Biomedical Watch · Maladie de Huntington | Détail |
| α-Amyrin α-Amyrin — protection mitochondriale | Myopathies | Remyélinisation indirecte / réparation · Neuroprotection | Hypothèse exploratoire | Rapprochement automatique non validé | Protection mitochondriale et modulation de la pathologie Tau α-Amyrin est étudié dans Maladie d’Alzheimer et documente plusieurs axes biologiques pouvant être pertinents pour Myopa… | Rapprochement Biomedical Watch · Maladie d’Alzheimer | Détail |
| 4-octyl itaconate et dérivés Dérivés de l’itaconate — neuroinflammation | Myopathies | Neuroprotection · Immunomodulation | Hypothèse exploratoire | Rapprochement automatique non validé | Modulation immunométabolique de la microglie 4-octyl itaconate et dérivés est étudié dans Sclérose latérale amyotrophique et documente plusieurs axes biologiques po… | Rapprochement Biomedical Watch · Sclérose latérale amy… | Détail |
| Thonningianin A Thonningianin A — neuroprotection anti-amyloïde | Myopathies | Neuroprotection | Hypothèse exploratoire | Rapprochement automatique non validé | Modulation du stress oxydatif et des protéines associées à Alzheimer Thonningianin A est étudié dans Maladie d’Alzheimer et documente plusieurs axes biologiques pouvant être pertinents pou… | Rapprochement Biomedical Watch · Maladie d’Alzheimer | Détail |
| Vutiglabridin Vutiglabridin — autophagie et PON2 | Myopathies | Neuroprotection | Hypothèse exploratoire | Rapprochement automatique non validé | Activation de PON2 et stimulation de l’autophagie Vutiglabridin est étudié dans Maladie d’Alzheimer et documente plusieurs axes biologiques pouvant être pertinents pour… | Rapprochement Biomedical Watch · Maladie d’Alzheimer | Détail |