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 |
|---|---|---|---|---|---|---|---|
| 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 |
|---|---|---|---|---|---|---|---|
| The efficacy of injecting a distillate of the pitcher plant (Sarracen… Mepivacaine Hydrochloride · PMID 38145776 | Myopathies | Traitement symptomatique | Publication | PubMed | Voir la publication source This study aimed to determine the efficacy of instilling extract of the pitcher plant around the palmar digital nerves… | Journal of equine veterinary science 2026-08-16 13:25:54 | Source |
| Comparison between Perineural and Intravenous Dexamethasone on Durati… Lidocaine Hydrochloride · PMID 41574836 | Myopathies | Traitement symptomatique | Publication | PubMed | Voir la publication source A disadvantage of regional blocks is their limited period of efficacy. One of the medications that has been found to ha… | Annals of African medicine 2026-08-16 13:25:53 | Source |
| Topical delivery of Lidocaine using spicules for local anesthesia. Lidocaine Hydrochloride · PMID 41402578 | Myopathies | Traitement symptomatique · Thérapie génique | Publication | PubMed | Voir la publication source Topical anesthesia offers a painless alternative to injections but is limited by the low skin permeability of local ane… | Drug delivery and translational research 2026-08-16 13:25:53 | Source |
| Efficacy of dry needling in reducing spasticity after stroke: A syste… Dry Needling · PMID 42603654 | Myopathies | Traitement symptomatique | Publication | PubMed | Voir la publication source To assess the effectiveness of dry needling (DN) in reducing spasticity among post-stroke patients through meta-analysi… | Complementary therapies in medicine 2026-08-16 13:25:50 | Source |
| Interventional Procedures in Sports. Dry Needling · PMID 42547150 | Myopathies | Immunomodulation · Traitement symptomatique · Thérapie génique | Publication | PubMed | Voir la publication source Image-guided percutaneous procedures are increasingly important in managing sports-related overuse and degenerative inj… | Radiologic clinics of North America 2026-08-16 13:25:50 | Source |
| Operational taxonomy as a determinant of classification stability in… Dry Needling · PMID 42235437 | Myopathies | Neuroprotection | Publication | PubMed | Voir la publication source Reproducibility in rehabilitation evidence synthesis is influenced not only by search strategy and adjudication archite… | International journal of medical informatics 2026-08-16 13:25:50 | Source |
| CACNA1C -Related Channelopathy Presenting With Adult-Onset Combined D… Xeomin Injectable Product · PMID 41978253 | Myopathies | Traitement symptomatique | Publication | PubMed | Voir la publication source Pathogenic variants in CACNA1C have been associated with cardiac arrhythmias and neurodevelopmental disorders, occasion… | American journal of medical genetics. Part A 2026-08-16 13:25:47 | Source |
| Late recovery of responsiveness after intra-thecal baclofen pump impl… infusion pump for intrathecal baclofen, Synchromed, Medtronic · PMID 31267188 | Myopathies | Traitement symptomatique | Publication | PubMed | Voir la publication source Patients with a prolonged disorder of consciousness (DoC) may present with severe spasticity and diffuse pain, which mi… | Acta neurochirurgica 2026-08-16 13:25:46 | Source |
| Wearable sensors in paediatric neurology. Syde · PMID 39888848 | Myopathies | Ralentissement de la progression · Thérapie génique | Publication | PubMed | Voir la publication source Wearable sensors have the potential to transform diagnosis, monitoring, and management of children who have neurologica… | Developmental medicine and child neurology 2026-08-16 13:25:41 | Source |
| Effects of carnosine supplementation on physical endurance: a placebo… Physical therapy · PMID 42308284 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Carnosine (β-alanine-L-histidine) is an endogenous dipeptide found in abundance in highly metabolic tissues such as ske… | Journal of the International Society of Sports Nutriti… 2026-08-16 13:25:37 | Source |
| Learning from Inuit perspectives on timimut ikajuqsivik/rehabilitatio… Physical therapy · PMID 42409750 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source The practice approaches and evidence base of timimut ikajuqtiit/rehabilitation professionals working with children are… | International journal of circumpolar health 2026-08-16 13:25:37 | Source |
| Instant analgesic effect of radial extracorporeal shock wave therapy… sham shockwave therapy · PMID 32046753 | Myopathies | Immunomodulation · Traitement symptomatique | Publication | PubMed | Voir la publication source Primary dysmenorrhoea (PDM) is defined as a series of pain-dominated symptoms during and after menstruation without org… | Trials 2026-08-16 13:25:36 | Source |
| Association between radial extracorporeal shock wave therapy combined… Radial extracorporeal shock wave therapy (rESWT) · PMID 42264811 | Myopathies | Traitement symptomatique | Publication | PubMed | Voir la publication source Few studies have evaluated the combined effects of radial extracorporeal shock wave therapy (rESWT) and physical therap… | Journal of bodywork and movement therapies 2026-08-16 13:25:33 | Source |
| [Translated article] Short- and mid-term efficacy of extracorporeal s… Radial extracorporeal shock wave therapy (rESWT) · PMID 42023623 | Myopathies | Traitement symptomatique · Thérapie génique | Publication | PubMed | Voir la publication source Trigger finger is a common condition managed through various conservative approaches. This study aimed to compare the s… | Revista espanola de cirugia ortopedica y traumatologia 2026-08-16 13:25:33 | Source |
| A matrix-adapted RNA extraction strategy for targeted RT-qPCR analysi… RNA extraction · PMID 42548905 | Myopathies | Thérapie génique | Publication | PubMed | Voir la publication source Human amniotic membrane (HAM) is a promising scaffold for periodontal tissue engineering, supporting favorable cellular… | Regenerative therapy 2026-08-16 13:25:30 | Source |
| Optimizing protocols for microRNA profiling of infant and toddler sto… RNA extraction · PMID 41715892 | Myopathies | Thérapie génique | Publication | PubMed | Voir la publication source Despite growing interest in profiling microRNAs (miRNAs) in infant and toddler stool, no studies have compared protocol… | RNA biology 2026-08-16 13:25:30 | Source |
| Salivary RANKL and OPG gene expression quantification during intermax… RNA extraction · PMID 42318067 | Myopathies | Thérapie génique | Publication | PubMed | Voir la publication source This protocol describes a non-invasive workflow for quantifying salivary RANKL and OPG gene expression in orthodontic p… | MethodsX 2026-08-16 13:25:30 | Source |
| Human neutrophils synthesize thrombomodulin that does not promote thr… Monocytes and megacaryocytes culture and RNA extraction · PMID 1325211 | Myopathies | Immunomodulation · Thérapie génique | Publication | PubMed | Voir la publication source Thrombomodulin (TM) is a surface glycoprotein that forms a 1:1 complex with thrombin, thereby interacting to form the b… | Blood 2026-08-16 13:25:29 | Source |
| The feasibility and acceptability of outdoor sessions as an add-on of… Effectiveness of Multicomponent Treatment for Fibromyalgia (FIBROWALK) · PMID 40316398 | Myopathies | Traitement symptomatique | Publication | PubMed | Voir la publication source The given text describes a pilot randomized controlled trial aimed at evaluating the feasibility and acceptability of o… | Reumatologia clinica 2026-08-16 13:25:17 | Source |
| A randomized placebo-controlled phase 3 trial of an antisense oligonu… Drisapersen · PMID 29203355 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source This 48-week, randomized, placebo-controlled phase 3 study (DMD114044; NCT01254019) evaluated efficacy and safety of su… | Neuromuscular disorders : NMD 2026-08-16 13:25:16 | 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 |