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
Filtrer toute la vue
Chaque choix s’applique immédiatement aux quatre sections. Le filtre « type de SEP » n’apparaît que lorsque la sclérose en plaques est sélectionnée.
1028 résultats correspondants
· 46 visibles sur cette page
dans le périmètre Myopathies
Niveau clinique
Études et traitements 434
Interventions, programmes et essais cliniques rattachés au périmètre sélectionné.
| Molécule / élément | Maladie / population | Objectif | Phase / stade | Statut / preuve | Mécanisme / résumé | Source | Détail |
|---|---|---|---|---|---|---|---|
| Adult and Juvenile Myositis NCT00017914 · NCT00017914 | Myopathies | Ralentissement de la progression · Immunomodulation · Traitement symptomatique… | À vérifier | RECRUITING | This study will evaluate subjects with adult- and childhood-onset myositis… À vérifier | ClinicalTrials.gov 2026-08-16 21:21:43 | Détail |
| Botulinum toxin type A NCT05720065 · Peripheral TMD Pain Mechanisms and the Effect by Botulinum Toxin A | Myopathies | Traitement symptomatique | Phase 2 | RECRUITING | The main purpose of this study is to determine which is the best dose of a… À vérifier | ClinicalTrials.gov 2026-08-16 21:21:43 | Détail |
| CovExc — Muscular Rehabilitation by Eccentric Exercise After Severe C… NCT04649086 · NCT04649086 | Myopathies | À vérifier | Non applicable | TERMINATED | With the COVID-19 pandemic, the number of patients to be treated in rehabi… À vérifier | ClinicalTrials.gov 2026-08-16 21:21:43 | Détail |
| Effectiveness of Conservative Interventions in the Treatment of Trigg… NCT06296017 · NCT06296017 | Myopathies | À vérifier | Non applicable | RECRUITING | There is no study in the literature comparing the effectiveness of ESWT an… À vérifier | ClinicalTrials.gov 2026-08-16 21:21:43 | Détail |
| Electrophysiological measurement of reflex nociceptive flexion thresh… NCT04624581 · FIbromyalgia anD GenetIcs Subgroups (FIDGIS) | Myopathies | Traitement symptomatique | Non applicable | COMPLETED | Fibromyalgia syndrome (FS) is characterized by widespread pain and affect… À vérifier | ClinicalTrials.gov 2026-08-16 21:21:43 | Détail |
| Fascial Distortion Model NCT06627855 · Effectiveness of the Fascial Distortion Model in Temporomandibular Disorders: A… | Myopathies | Ralentissement de la progression · Traitement symptomatique | Non applicable | RECRUITING | The aim of the study was to investigate the effectiveness of Manual Therap… À vérifier | ClinicalTrials.gov 2026-08-16 21:21:43 | Détail |
| Intravenously administered pooled human immunoglobulin (IVIG) NCT06599697 · The MIGHT Trial - An Exploratory Clinical Trial of IVIG in Anti-HMGCR Immune Med… | Myopathies | À vérifier | Phase 2 | RECRUITING | This is a randomized, placebo-controlled, double blinded phase 2 explorato… À vérifier | ClinicalTrials.gov 2026-08-16 21:21:43 | Détail |
| LOLA — Effectiveness of Health Education Program on Musculoskeletal P… NCT07160478 · NCT07160478 | Myopathies | Traitement symptomatique | Non applicable | COMPLETED | Musculoskeletal pain during childhood can negatively affect school attenda… À vérifier | ClinicalTrials.gov 2026-08-16 21:21:43 | Détail |
| Prospective, Longitudinal, Observational Registry of Adult Patients W… NCT05596539 · NCT05596539 | Myopathies | À vérifier | À vérifier | RECRUITING | The purpose of this study is to assess medical events during follow-up of… À vérifier | ClinicalTrials.gov 2026-08-16 21:21:43 | Détail |
| Real radial extracorporeal shock wave therapy (rESWT) NCT07476690 · CP-rESWT-FREQ — Effect of rESWT Session Frequency on Spasticity and Function Ass… | Myopathies | Traitement symptomatique | Non applicable | COMPLETED | Spasticity is a major contributor to functional limitations in children wi… À vérifier | ClinicalTrials.gov 2026-08-16 21:21:43 | Détail |
| SeMAFor — Magnesium, Stress and Fibromyalgia NCT03887000 · NCT03887000 | Myopathies | Traitement symptomatique | Non applicable | COMPLETED | Fibromyalgia affects an average of 2% of the French population. Data from… À vérifier | ClinicalTrials.gov 2026-08-16 21:21:43 | Détail |
| AOC 1020 NCT05747924 · FORTITUDE — Phase 1/2 Study of AOC 1020 in Participants With Facioscapulohumeral… | Myopathies | À vérifier | Phase 1/2 | COMPLETED | A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluat… À vérifier | ClinicalTrials.gov 2026-08-16 19:04:32 | Détail |
| BMN 351 NCT06280209 · A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Phar… | Myopathies | À vérifier | Phase 1/2 | ACTIVE_NOT_RECRUITING | This open-label extension study aims to evaluate the long-term safety and… À vérifier | ClinicalTrials.gov 2026-08-16 19:04:32 | Détail |
| EPI-321 NCT06907875 · FSHD — A First-in-human Study of EPI-321 in Facioscapulohumeral Muscular Dystrop… | Myopathies | Thérapie génique | Phase 1/2 | ACTIVE_NOT_RECRUITING | The goal of this clinical trial is to learn how safe and tolerable EPI-321… À vérifier | ClinicalTrials.gov 2026-08-16 19:04:32 | Détail |
| GNR-097 NCT07673809 · A Study to Evaluate the Tolerability, Safety and Efficacy of GNR-097 Gene Therap… | Myopathies | Thérapie génique | Phase 1/2 | RECRUITING | The study will evaluate the tolerability, safety and efficacy of gene ther… À vérifier | ClinicalTrials.gov 2026-08-16 19:04:32 | Détail |
| PBGENE-DMD (IV) NCT07429240 · PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular… | Myopathies | À vérifier | Phase 1/2 | RECRUITING | The purpose of this Phase 1/2a trial is to evaluate the safety, tolerabili… À vérifier | ClinicalTrials.gov 2026-08-16 19:04:32 | 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 |
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 588
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 |
|---|---|---|---|---|---|---|---|
| Clinical Practice Recommendations for Non-Dermatologists on the Diagn… Medical treatment · PMID 42590188 | Myopathies | Immunomodulation · Traitement symptomatique | Publication | PubMed | Voir la publication source Generalized pustular psoriasis (GPP) is a rare, potentially life-threatening, chronic cutaneous inflammatory disease ch… | Journal of clinical medicine 2026-08-13 15:03:56 | Source |
| Mechanisms Underlying the Benefits of Coffee in Postoperative Ileus. Medical treatment · PMID 42588126 | Myopathies | Immunomodulation | Publication | PubMed | Voir la publication source Postoperative ileus (POI) is a motility dysfunction associated with inflammation in the gastrointestinal (GI) tract aft… | Nutrients 2026-08-13 15:03:56 | Source |
| Prednisone, not vamorolone, suppresses novel serum bone and cartilage… Vamorolone · PMID 42547787 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Corticosteroids are among the most highly prescribed drugs. Chronic treatment with corticosteroids causes osteopenia, a… | Scientific reports 2026-08-13 15:03:55 | Source |
| Vagus nerve stimulation as an add-on therapy in patients with epileps… Vagus nerve stimulation · PMID 42581390 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Vagus nerve stimulation (VNS) has been proven as an effective and safe adjunct therapy for epilepsy, but real-world evi… | Acta epileptologica 2026-08-13 15:03:55 | Source |
| Vamorolone for Duchenne Muscular Dystrophy: A Cross-Trial Efficacy Co… Vamorolone · PMID 42531535 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Résumé non disponible dans la base locale. | Neurology 2026-08-13 15:03:55 | Source |
| Remogliflozin Attenuates Thioacetamide-Induced Nephrotoxicity: Associ… SGLT2 inhibitor · PMID 42590936 | Myopathies | Neuroprotection · Immunomodulation | Publication | PubMed | Voir la publication source Thioacetamide (TAA) induces renal injury via oxidative stress and inflammation. Remogliflozin (Remo), an SGLT2 inhibito… | Toxicology mechanisms and methods 2026-08-13 15:03:54 | Source |
| SGLT2 Inhibitors and Outcomes in Elderly Patients With Chronic Kidney… SGLT2 inhibitor · PMID 42587349 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Sodium-glucose cotransporter 2 (SGLT2) inhibitors have emerged as a cornerstone therapy for chronic kidney disease (CKD… | Diabetes, obesity & metabolism 2026-08-13 15:03:54 | Source |
| Kidney outcomes and safety of sodium-glucose cotransporter-2 inhibito… SGLT-2 inhibitor · PMID 42586506 | Myopathies | Thérapie génique | Publication | PubMed | Voir la publication source Autosomal dominant polycystic kidney disease (ADPKD) is the most common hereditary kidney disease and among the leading… | Diabetes research and clinical practice 2026-08-13 15:03:53 | Source |
| AAV-mediated gene transfer of a novel microdystrophin ameliorates pat… RGX-202 · PMID 42003884 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Use of adeno-associated virus (AAV)-mediated transfer of functional microdystrophins to address Duchenne muscular dystr… | Molecular therapy. Nucleic acids 2026-08-13 15:03:52 | Source |
| Targeting the creatine transporter SLC6A8: Mechanisms and emerging th… RGX-202 · PMID 42297217 | Myopathies | Remyélinisation indirecte / réparation · Thérapie génique | Publication | PubMed | Voir la publication source Solute carrier family 6 member 8 (SLC6A8) is a Na- and Cl-dependent creatine transporter that mediates the transmembran… | Biochemical pharmacology 2026-08-13 15:03:52 | Source |
| AAV microdystrophin gene replacement therapy for Duchenne muscular dy… PF-06939926 · PMID 40817386 | Myopathies | Thérapie cellulaire · Thérapie génique | Publication | PubMed | Voir la publication source Duchenne muscular dystrophy (DMD) is caused by pathogenic sequence variants occurring in the DMD gene which lead to the… | Gene therapy 2026-08-13 15:03:50 | Source |
| Cardiac safety of fordadistrogene movaparvovec gene therapy in Duchen… PF-06939926 · PMID 40583273 | Myopathies | Ralentissement de la progression · Thérapie génique | Publication | PubMed | Voir la publication source Fordadistrogene movaparvovec (FM; PF-06939926) is a recombinant adeno-associated virus serotype-9 gene-replacement cons… | Molecular therapy : the journal of the American Societ… 2026-08-13 15:03:50 | Source |
| An Antibody-Oligonucleotide Conjugate for Myotonic Dystrophy Type 1. Del-desiran (AOC 1001) · PMID 41707138 | Myopathies | Thérapie génique | Publication | PubMed | Voir la publication source Myotonic dystrophy type 1 is a rare, dominantly inherited, progressive, disabling, neuromuscular disease that leads to… | The New England journal of medicine 2026-08-13 15:03:49 | Source |
| Effect of using knee extension aid for gait training in subacute hemi… Extension assist knee ankle foot orthosis (EA-KAFO) · PMID 42544336 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source [Purpose] In normal gait, the knee flexes approximately 60° during the swing phase, and the lower leg's inertial force… | Journal of physical therapy science 2026-08-13 15:03:49 | Source |
| Robot-Assisted Gait Training to Improve Gait Patterns in Two Adolesce… Extension assist knee ankle foot orthosis (EA-KAFO) · PMID 41332284 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Although the effectiveness of robot-assisted gait training (RAGT) in stroke has been reported, evidence in adolescents… | Physical & occupational therapy in pediatrics 2026-08-13 15:03:49 | Source |
| BMN 351-Induced Exon Skipping and Dystrophin Expression in Skeletal a… BMN 351 · PMID 39916519 | Myopathies | Thérapie génique | Publication | PubMed | Voir la publication source Duchenne muscular dystrophy (DMD) is caused by mutations of the gene that prevent the expression of functional dystroph… | Nucleic acid therapeutics 2026-08-13 15:03:48 | Source |
| Targeting a Novel Site in Exon 51 with Antisense Oligonucleotides Ind… BMN 351 · PMID 39916530 | Myopathies | Thérapie génique | Publication | PubMed | Voir la publication source Exon skipping with antisense oligonucleotides (ASOs) can correct disease-causing mutations of Duchenne muscular dystrop… | Nucleic acid therapeutics 2026-08-13 15:03:48 | Source |
| From design to clinic: Medicinal chemistry and pharmacology of approv… Givinostat · PMID 42546588 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Many diseases, including cancer, are characterized by increased or decreased expression of specific genes. These change… | European journal of medicinal chemistry 2026-08-13 15:03:47 | Source |
| Real-world safety profile of givinostat: an early post-marketing phar… Givinostat · PMID 42494524 | Myopathies | Ralentissement de la progression | Publication | PubMed | Voir la publication source Givinostat is a novel histone deacetylase inhibitor, which was approved by the US Food and Drug Administration (FDA) in… | Frontiers in pharmacology 2026-08-13 15:03:47 | Source |
| Bone marrow mesenchymal stem cell exosomes in osteonecrosis: patholog… Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC) · PMID 42550377 | Myopathies | Traitement symptomatique · Thérapie cellulaire | Publication | PubMed | Voir la publication source Osteonecrosis is bone death caused by disrupted blood supply. It often leads to bone collapse and joint failure. Curren… | Molecular biology reports 2026-08-13 15:03:46 | 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 |