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 |
|---|---|---|---|---|---|---|---|
| Track-DM — An Ambispective Natural History Study in Myotonic Dystroph… NCT07732439 · Track-DM — An Ambispective Natural History Study in Myotonic Dystrophy Patients… | Myopathies | Traitement symptomatique | À vérifier | NOT_YET_RECRUITING | This natural history observational study is being conducted to follow pati… À vérifier | ClinicalTrials.gov 2026-08-13 14:17:36 | Détail |
| Vamorolone NCT06564974 · DMD-001 SUMMIT — Registry Study to Observe Long-term Safety of Vamorolone (AGAMR… | Myopathies | À vérifier | À vérifier | RECRUITING | The goal of this study is to collect additional information on the safety… À vérifier | ClinicalTrials.gov 2026-08-13 14:17:36 | Détail |
| A Clinical Trial to Evaluate the Effects of Peptide Gummies on Marker… NCT07752381 · NCT07752381 | Myopathies | Immunomodulation | Non applicable | COMPLETED | This study will evaluate the effects of Parlay Recover peptide gummies (co… À vérifier | ClinicalTrials.gov 2026-08-13 14:17:35 | Détail |
| Myofascial Release and Positional Release Techniques in Unilateral Tr… NCT07515352 · NCT07515352 | Myopathies | Ralentissement de la progression · Traitement symptomatique | Non applicable | COMPLETED | Unilateral trapezitis is characterized by upper trapezius myofascial pain… À vérifier | ClinicalTrials.gov 2026-08-13 14:17:35 | Détail |
| Pimicotinib NCT07499362 · Study of Pimicotinib in Japanese Participants With Tenosynovial Giant Cell Tumor… | Myopathies | Immunomodulation | Phase 2 | RECRUITING | The primary purpose of this study is to assess the tolerability, pharmacok… À vérifier | ClinicalTrials.gov 2026-08-13 14:17:35 | Détail |
| the — Eccentric vs Low-Load Blood Flow Restriction Training in Knee O… NCT07733518 · NCT07733518 | Myopathies | Ralentissement de la progression | Non applicable | RECRUITING | The aim of this randomized controlled trial is to find the effects of ecce… À vérifier | ClinicalTrials.gov 2026-08-13 14:17:35 | Détail |
| Yoomi Physical Therapy Software NCT05815017 · YOOMI: Effect of Gamified Physical Therapy Exercise Software on Inpatient Mobili… | Myopathies | À vérifier | Non applicable | COMPLETED | Patients admitted to the hospital often develop functional impairments due… À vérifier | ClinicalTrials.gov 2026-08-13 14:17:35 | Détail |
| 3 Year Follow up on ANO5 Patients NCT05206617 · NCT05206617 | Myopathies | Ralentissement de la progression | À vérifier | ACTIVE_NOT_RECRUITING | The aim of the study is to investigate progression in muscle affection in… À vérifier | ClinicalTrials.gov 2026-08-13 14:17:34 | Détail |
| ANO5 MRI — MRI-phenotyping of Patients With Pathogenic Anoctamin 5 Va… NCT05102799 · NCT05102799 | Myopathies | Thérapie génique | À vérifier | ACTIVE_NOT_RECRUITING | A large cohort of MRI scans from patients with pathogenic variants in the… À vérifier | ClinicalTrials.gov 2026-08-13 14:17:34 | Détail |
| Apitegromab NCT07435129 · Phase 2 Study Evaluating Apitegromab for the Treatment of FSHD | Myopathies | À vérifier | Phase 2 | RECRUITING | A randomized Phase 2 study to evaluate the efficacy and safety of apitegro… À vérifier | ClinicalTrials.gov 2026-08-13 14:17:34 | Détail |
| Botulinum toxin type A NCT07758322 · Efficacy of Focused Shock Wave Therapy Before and After Botulinum Toxin Injectio… | Myopathies | Traitement symptomatique | Non applicable | ACTIVE_NOT_RECRUITING | The main purpose of this study is to determine which is the best dose of a… À vérifier | ClinicalTrials.gov 2026-08-13 14:17:34 | Détail |
| Constant Current Stimulator NCT07715006 · Dorsal Root Electrical Stimulation in Children With Cerebral Palsy | Myopathies | Traitement symptomatique | Non applicable | RECRUITING | The goal of this clinical trial is to evaluate whether dorsal root electri… À vérifier | ClinicalTrials.gov 2026-08-13 14:17:34 | Détail |
| Conventional Stabilization Splints NCT07760428 · Comparison of Conventional,3D Printed and Novel Hybrid Stabilization Splints in… | Myopathies | Traitement symptomatique | Non applicable | NOT_YET_RECRUITING | Occlusal splint therapy is a well-established conservative approach for th… À vérifier | ClinicalTrials.gov 2026-08-13 14:17:34 | Détail |
| delpacibart zotadirsen NCT07250737 · Managed Access Program for Del-zota in Participants With DMD Mutations Amenable… | Myopathies | À vérifier | À vérifier | AVAILABLE | The purpose of this Managed Access Program is to allow access to delpaciba… À vérifier | ClinicalTrials.gov 2026-08-13 14:17:34 | Détail |
| DNA Methylation in PMR NCT07748195 · NCT07748195 | Myopathies | À vérifier | À vérifier | NOT_YET_RECRUITING | Polymyalgia Rheumatica (PMR) is treated with corticosteroids; however, lon… À vérifier | ClinicalTrials.gov 2026-08-13 14:17:34 | Détail |
| Dry Needling NCT07321483 · Reflex Therapy of Temporomandibular Dysfunctions | Myopathies | Traitement symptomatique | Non applicable | RECRUITING | This randomized clinical trial will aim to compare the effectiveness of dr… À vérifier | ClinicalTrials.gov 2026-08-13 14:17:34 | Détail |
| Dual-task cycling NCT07580365 · VirtualPark_Pediatric | Myopathies | À vérifier | Non applicable | RECRUITING | The goal of this clinical trial is to assess the effects of a dual-task, m… À vérifier | ClinicalTrials.gov 2026-08-13 14:17:34 | Détail |
| Effects of Curcumin-Piperine Supplementation in Fibromyalgia NCT07753018 · NCT07753018 | Myopathies | Neuroprotection · Immunomodulation · Traitement symptomatique | Phase 2 | NOT_YET_RECRUITING | The goal of this clinical trail is to evaluate the effectiveness and safet… À vérifier | ClinicalTrials.gov 2026-08-13 14:17:34 | Détail |
| Effects of Pilates and Desk-Based Exercise on Office Workers NCT07763925 · NCT07763925 | Myopathies | Traitement symptomatique | Non applicable | COMPLETED | This study evaluated whether adding Pilates exercises to a desk-based exer… À vérifier | ClinicalTrials.gov 2026-08-13 14:17:34 | Détail |
| EXCEL — A Study to Check Liver Health in Boys With XLMTM, a Serious G… NCT06581146 · NCT06581146 | Myopathies | Traitement symptomatique · Thérapie génique | À vérifier | RECRUITING | XLMTM (X-linked myotubular myopathy) is a serious genetic muscle condition… À vérifier | ClinicalTrials.gov 2026-08-13 14:17:34 | 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 |