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 |
|---|---|---|---|---|---|---|---|
| AB-1003 dose level 1 NCT05230459 · A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With… | Myopathies | À vérifier | Phase 1/2 | ACTIVE_NOT_RECRUITING | The purpose of this study is to evaluate the safety and tolerability of a… À vérifier | ClinicalTrials.gov 2026-09-01 09:22:28 | Détail |
| Alternate-day prednisolone NCT07787689 · Intermittent vs Alternate Daily Regimen of Prednisolone in Ambulatory Boys With… | Myopathies | Thérapie génique | Non applicable | NOT_YET_RECRUITING | The alternate-day regimen is hypothesized to reduce hypothalamic-pituitary… À vérifier | ClinicalTrials.gov 2026-09-01 09:22:28 | Détail |
| ELEVIDYS NCT07542314 · ENHANCE — Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Particip… | Myopathies | À vérifier | Phase 4 | NOT_YET_RECRUITING | The primary objective of this study is to evaluate acute liver injury (ALI… À vérifier | ClinicalTrials.gov 2026-09-01 09:22:28 | Détail |
| Evaluating VM100 Nutritional Supplement for Improving Quality of Life… NCT07766980 · NCT07766980 | Myopathies | Traitement symptomatique | Non applicable | RECRUITING | This pilot study will investigate the potential efficacy of VM100, a nutri… À vérifier | ClinicalTrials.gov 2026-09-01 09:22:28 | Détail |
| force gravity stimulation NCT07780435 · Whole-body Vibration Versus Gravity Force Stimulation on Postural Stability in C… | Myopathies | À vérifier | Non applicable | COMPLETED | this study aimed to mention Does whole-body vibration training or gravity-… À vérifier | ClinicalTrials.gov 2026-09-01 09:22:28 | Détail |
| International GNE Myopathy Patient Registry NCT04009226 · NCT04009226 | Myopathies | Ralentissement de la progression | À vérifier | COMPLETED | GNE myopathy, an ultra-rare disease, is a severe progressive myopathy that… À vérifier | ClinicalTrials.gov 2026-09-01 09:22:28 | Détail |
| Investigational Use of Neuromuscular Ultrasound NCT05237973 · NCT05237973 | Myopathies | Traitement symptomatique · Thérapie génique | À vérifier | RECRUITING | Background: Current techniques used to measure the health and function of… À vérifier | ClinicalTrials.gov 2026-09-01 09:22:28 | Détail |
| Observation Study in Patients Age 0-5 Years With LAMA2-related Congen… NCT06503367 · NCT06503367 | Myopathies | Thérapie génique | À vérifier | RECRUITING | The goal of this observational study is to understand how young children w… À vérifier | ClinicalTrials.gov 2026-09-01 09:22:28 | Détail |
| TAS-205 [Ambulatory Cohort] [Non-ambulatory Cohort] NCT04587908 · A Phase 3 Study of TAS-205 in Patients With Duchenne Muscular Dystrophy（REACH-DM… | Myopathies | À vérifier | Phase 3 | COMPLETED | The purpose of this study is to evaluate the efficacy and safety of TAS-20… À vérifier | ClinicalTrials.gov 2026-09-01 09:22:28 | Détail |
| The Baby Duchenne Study: Characterizing Developmental and Clinical Ou… NCT07092540 · NCT07092540 | Myopathies | À vérifier | À vérifier | RECRUITING | The aim of the BABY DUCHENNE study is to evaluate the natural history and… À vérifier | ClinicalTrials.gov 2026-09-01 09:22:28 | Détail |
| The United Kingdom Facioscapulohumeral Muscular Dystrophy Patient Reg… NCT04001582 · NCT04001582 | Myopathies | À vérifier | À vérifier | RECRUITING | Facioscapulohumeral Dystrophy (FSHD) is the third most common form of neur… À vérifier | ClinicalTrials.gov 2026-09-01 09:22:28 | Détail |
| VX-670 NCT06185764 · Galileo — A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystro… | Myopathies | À vérifier | Phase 1/2 | ACTIVE_NOT_RECRUITING | The purpose of the study is to evaluate the long-term safety and tolerabil… À vérifier | ClinicalTrials.gov 2026-09-01 09:22:28 | Détail |
| Active full-body stimulation NCT07132775 · Evaluation of the Safety and Efficacy of a Full-Body Electrostimulation Garment… | Myopathies | Traitement symptomatique | Non applicable | RECRUITING | The purpose of this study is to to explore the safety and efficacy of the… À vérifier | ClinicalTrials.gov 2026-09-01 09:22:27 | Détail |
| A Natural History Study of Patients With GNE Myopathy and GNE-Related… NCT01417533 · NCT01417533 | Myopathies | Traitement symptomatique · Thérapie génique | À vérifier | COMPLETED | Background: \- GNE Myopathy is a disease that causes walking difficulties… À vérifier | ClinicalTrials.gov 2026-09-01 09:22:27 | Détail |
| Antidepressant NCT04747314 · TNA-LBP — Treating Negative Affect in Low Back Pain Patients | Myopathies | Traitement symptomatique | Phase 2 | COMPLETED | This study will examine how the use of antidepressant, physical therapy, a… À vérifier | ClinicalTrials.gov 2026-09-01 09:22:27 | Détail |
| Deramiocel (CAP-1002) NCT05126758 · HOPE-3 — A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patie… | Myopathies | Thérapie cellulaire | Phase 3 | ACTIVE_NOT_RECRUITING | HOPE-3 is a two cohort, Phase 3, multi-center, randomized, double-blind, p… À vérifier | ClinicalTrials.gov 2026-09-01 09:22:27 | Détail |
| FOPal — A Study to Document and to Further Describe Long-term Safety… NCT06089616 · NCT06089616 | Myopathies | Traitement symptomatique | À vérifier | RECRUITING | The participants in this registry study will have fibrodysplasia ossifican… À vérifier | ClinicalTrials.gov 2026-09-01 09:22:27 | Détail |
| Metformin NCT00720161 · Metformin in Children With Motor Deficit | Myopathies | À vérifier | Non applicable | COMPLETED | The goal of this clinical trial is to Evaluating Metformin Efficacy and Sa… À vérifier | ClinicalTrials.gov 2026-09-01 09:22:27 | Détail |
| MOTIVATE_IIR — MOTIVATE to Improve Outcomes for Older Veterans With M… NCT05220202 · NCT05220202 | Myopathies | Traitement symptomatique | Non applicable | COMPLETED | The proposed study seeks to evaluate effectiveness, implementation process… À vérifier | ClinicalTrials.gov 2026-09-01 09:22:27 | Détail |
| Nerandomilast NCT07789015 · VERANDA™-IIM — A Study to Understand How Nerandomilast Works in People With Acti… | Myopathies | Immunomodulation · Traitement symptomatique | Phase 3 | NOT_YET_RECRUITING | This study is open to adults aged 18 and older who have a condition known… À vérifier | ClinicalTrials.gov 2026-09-01 09:22:27 | 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 |
|---|---|---|---|---|---|---|---|
| Liver health in myotubular and centronuclear myopathies: a patient-dr… CMDPROS — Congenital Muscle Disease Study of Patient and Family Reported Medical Information ·… | Myopathies | Traitement symptomatique | Publication | PubMed | Voir la publication source Liver health issues in X-linked myotubular myopathy and centronuclear myopathies have historically been under-recognize… | Neuromuscular disorders : NMD 2026-08-13 15:02:49 | Source |
| Six novel SACS mutations expand the autosomal recessive spastic ataxi… CMDPROS — Congenital Muscle Disease Study of Patient and Family Reported Medical Information ·… | Myopathies | Traitement symptomatique | Publication | PubMed | Voir la publication source BACKGROUND: The clinical spectrum of autosomal recessive spastic ataxia of Charlevoix–Saguenay (ARSACS) in Asian popula… | Orphanet journal of rare diseases 2026-08-13 15:02:49 | Source |
| Neuromorphic Devices and Computing for Sensing, Memory, and Control. Stem Cell · PMID 42591065 | Myopathies | Thérapie cellulaire | Publication | PubMed | Voir la publication source Neuromorphic devices are bioinspired electronic systems that mimic key structures and functions of the nervous system,… | Advanced science (Weinheim, Baden-Wurttemberg, Germany) 2026-08-13 15:02:48 | Source |
| Effect of Instrument-Assisted Soft Tissue Mobilization Combined With… Effect of Phonophoresis on Trigger Points · PMID 41206797 | Myopathies | Ralentissement de la progression · Traitement symptomatique | Publication | PubMed | Voir la publication source The purpose of this study was to investigate the effects of instrument-assisted soft tissue mobilization (IASTM) and li… | Journal of manipulative and physiological therapeutics 2026-08-13 15:02:47 | Source |
| Platelet rich plasma therapy versus other modalities for treatment of… Effect of Phonophoresis on Trigger Points · PMID 38395675 | Myopathies | Traitement symptomatique | Publication | PubMed | Voir la publication source Plantar fasciitis (PF) is the most common cause of heel pain in adults. There are numerous non-operative treatments ava… | Foot and ankle surgery : official journal of the Europ… 2026-08-13 15:02:47 | Source |
| Clinical effect of kinesio taping combined with dry needling to myofa… Dry Needling for Myofascial Pain Syndrome · PMID 42545155 | Myopathies | Ralentissement de la progression · Traitement symptomatique | Publication | PubMed | Voir la publication source Shoulder-hand syndrome (SHS) is a common complication after stroke, characterized by pain, edema, and functional impair… | European journal of physical and rehabilitation medici… 2026-08-13 15:02:46 | Source |
| Comparative effects of low-level laser acupuncture and dry needling o… Dry Needling for Myofascial Pain Syndrome · PMID 42430011 | Myopathies | Ralentissement de la progression · Traitement symptomatique | Publication | PubMed | Voir la publication source The purpose of this study was to compare the effectiveness of Low-Level Laser Acupuncture with Dry Needling along with… | Lasers in medical science 2026-08-13 15:02:46 | Source |
| Physical therapy interventions for awake and sleep bruxism: What has… Splint Therapy · PMID 42497348 | Myopathies | Ralentissement de la progression · Traitement symptomatique | Publication | PubMed | Voir la publication source The objective of this systematic review was to summarize the physiotherapy (PT) treatments used over the last few decad… | Dental and medical problems 2026-08-13 15:02:45 | Source |
| Volume of Treatment of Temporomandibular Disorders in Adults-A Nation… Splint Therapy · PMID 42449475 | Myopathies | Traitement symptomatique | Publication | PubMed | Voir la publication source Temporomandibular disorders (TMD) present diverse signs and symptoms and often require clinical evaluation. To investig… | Journal of oral rehabilitation 2026-08-13 15:02:45 | Source |
| A Phase 1, Double-Blind, Placebo-Controlled Trial of Sevasemten (EDG-… Sevasemten Dose 2 · PMID 40452637 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Sevasemten (EDG-5506) is an orally administered, investigational small molecule that selectively modulates fast muscle… | Muscle & nerve 2026-08-13 15:02:43 | Source |
| Effects of sevasemten (EDG-5506) on safety, biomarkers, and functiona… Sevasemten Dose 1 · PMID 42308588 | Myopathies | Immunomodulation | Publication | PubMed | Voir la publication source Sevasemten (EDG-5506) is an orally administered, investigational small molecule that selectively modulates fast muscle… | EBioMedicine 2026-08-13 15:02:42 | Source |
| Human Disposition, Metabolism, and Excretion of Sevasemten (EDG-5506)… Sevasemten Dose 1 · PMID 42394192 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Sevasemten (EDG-5506) is a novel, orally bioavailable, investigational small molecule designed to selectively modulate… | Journal of clinical pharmacology 2026-08-13 15:02:42 | Source |
| Airway Clearance in Neuromuscular Disease. Lung Volume Recruitment (LVR) · PMID 39095145 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source High-quality respiratory care and airway clearance is essential for people with neuromuscular disease (pwNMD) as respir… | Sleep medicine clinics 2026-08-13 15:02:40 | Source |
| Effects of Thermal Aging and Subsequent Autoclave Treatment on the Ph… (-)-Epicatechin · PMID 42588794 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source This study evaluated the effects of aging and subsequent autoclave treatment on the proton nuclear magnetic resonance (… | Plants (Basel, Switzerland) 2026-08-13 15:02:40 | Source |
| Integrated Transcriptomic and Metabolomic Profiling Reveals the Invol… (-)-Epicatechin · PMID 42588842 | Myopathies | Thérapie génique | Publication | PubMed | Voir la publication source Sorghum seeds accumulate substantial amounts of condensed tannins (CTs), which are also referred to as proanthocyanidin… | Plants (Basel, Switzerland) 2026-08-13 15:02:40 | Source |
| Patterns of Adherence to Lung Volume Recruitment Therapy Amongst Indi… Lung Volume Recruitment (LVR) · PMID 41147267 | Myopathies | Traitement symptomatique | Publication | PubMed | Voir la publication source Duchenne muscular dystrophy (DMD) is associated with impaired airway clearance and chest wall restriction, treated with… | Pediatric pulmonology 2026-08-13 15:02:40 | Source |
| Cardiac Myosin Activators in Heart Failure: Experimental Advances Ami… danicamtiv · PMID 41858918 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Heart failure remains a leading cause of morbidity and mortality despite advances in neurohormonal and device-based the… | Drug design, development and therapy 2026-08-13 15:02:38 | Source |
| The role of cardiac myosin activators in dilated cardiomyopathy pharm… danicamtiv · PMID 42212862 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Résumé non disponible dans la base locale. | Expert opinion on pharmacotherapy 2026-08-13 15:02:38 | Source |
| Local anesthetics and antibiotics display synergistic and antagonisti… Mepivacaine Hydrochloride · PMID 39970538 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source To investigate the in vitro efficacy of antibiotics (amikacin, ceftiofur, and gentamicin) in combination with local ane… | American journal of veterinary research 2026-08-13 15:02:37 | Source |
| Solvent-Driven Enrichment and Multiplex Analysis of Local Anesthetics… Mepivacaine Hydrochloride · PMID 40286164 | Myopathies | Traitement symptomatique | Publication | PubMed | Voir la publication source Local anesthetics have been widely used in clinical analgesia due to their ability to provide effective regional pain m… | Molecules (Basel, Switzerland) 2026-08-13 15:02:37 | 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 |