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.
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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 |
|---|---|---|---|---|---|---|---|
| Semaglutide (Rybelsus®) NCT07642635 · Glucagon-Like Peptide-1 Receptor Agonists to Attenuate Metabolic Risk in Individ… | Myopathies | Traitement symptomatique | Phase 1/2 | NOT_YET_RECRUITING | Duchenne Muscular Dystrophy (DMD) is a rare, genetic disease that leads to… À vérifier | ClinicalTrials.gov 2026-08-16 21:21:44 | Détail |
| Skeletal muscle biopsy (residual specimen) NCT06101940 · Chinese Multicenter Clinical Outcome Cohort Study of Myotonic Dystrophy Type 1 (… | Myopathies | Ralentissement de la progression | À vérifier | ENROLLING_BY_INVITATION | Myotonic dystrophy type 1 (DM1) is an autosomal dominant multisystem disor… À vérifier | ClinicalTrials.gov 2026-08-16 21:21:44 | Détail |
| TODDLER — A Remote Study Using Technology to Assess Outcomes in DMD NCT07423026 · NCT07423026 | Myopathies | À vérifier | À vérifier | RECRUITING | Every year, 100 boys are born in the UK with a rare muscle disease called… À vérifier | ClinicalTrials.gov 2026-08-16 21:21:44 | 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 |
| AZD5492 NCT06916806 · TITAN — A Study to Investigate Safety, Tolerability, Pharmacokinetics, and Pharm… | Lupus | Immunomodulation | Phase 1 | RECRUITING | The purpose of this study is to measure the safety, tolerability, PK, and… À vérifier | ClinicalTrials.gov 2026-08-16 21:21:41 | 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 |
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 |
|---|---|---|---|---|---|---|---|
| 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 |
| Subconjunctival Injection of Mesenchymal Stem Cells for Corneal Wound… Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC) · PMID 42454124 | Myopathies | Immunomodulation · Thérapie cellulaire | Publication | PubMed | Voir la publication source Previous studies in animal models have demonstrated that mesenchymal stem cells (MSCs) are beneficial for reconstructin… | Frontiers in medicine 2026-08-13 15:03:46 | Source |
| Development of a DUX4-targeting antibody oligonucleotide conjugate as… AOC-1020 · PMID 41994867 | Myopathies | Thérapie génique | Publication | PubMed | Voir la publication source Facioscapulohumeral muscular dystrophy (FSHD) is an autosomal dominant muscular disease in which genetic mutations acti… | Nucleic acids research 2026-08-13 15:03:45 | Source |
| Preclinical evaluation of INS1201 AAV9-micro-dystrophin via CSF admin… INS1201 · PMID 42137291 | Myopathies | Immunomodulation | Publication | PubMed | Voir la publication source INS1201 is an investigational adeno-associated virus 9-micro-dystrophin gene transfer therapy under development as a po… | Molecular therapy. Advances 2026-08-13 15:03:44 | 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 |