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 |
|---|---|---|---|---|---|---|---|
| Hyaluronic acid nanoparticle as drug delivery carrier for the treatme… Diclofenac 1% Topical · PMID 42578876 | Myopathies | Immunomodulation | Publication | PubMed | Voir la publication source BackgroundAllergic conjunctivitis is an inflammatory disorder of the conjunctiva triggered by allergens. Although topic… | Bio-medical materials and engineering 2026-08-13 15:01:42 | Source |
| Successful Management of Refractory Cannabinoid Hyperemesis Syndrome… Capsaicin Topical Cream · PMID 42491286 | Myopathies | Traitement symptomatique | Publication | PubMed | Voir la publication source Cannabinoid hyperemesis syndrome (CHS) is a debilitating disorder of chronic, heavy cannabis users characterized by cyc… | Clinical medicine insights. Case reports 2026-08-13 15:01:41 | Source |
| Fat Embolism Syndrome Following Elective Orthopedic Surgery in a Pati… Chronic Corticosteroid Therapy · PMID 42572732 | Myopathies | Immunomodulation | Publication | PubMed | Voir la publication source Duchenne muscular dystrophy (DMD) is associated with substantial perioperative morbidity due to progressive cardiopulmo… | Cureus 2026-08-13 15:01:38 | Source |
| Recalibrating Therapeutic Priorities for Duchenne Muscular Dystrophy:… Ataluren · PMID 42510866 | Myopathies | Thérapie génique | Publication | PubMed | Voir la publication source Duchenne muscular dystrophy (DMD) has, for the first time, several mutation-specific drugs in clinical use. Four exon-s… | Genes 2026-08-13 15:01:37 | Source |
| Modular intramedullary knee arthrodesis for chronic periprosthetic jo… Limb salvage · PMID 42585983 | Myopathies | Thérapie génique | Publication | PubMed | Voir la publication source Chronic periprosthetic joint infection (PJI) after total knee arthroplasty (TKA) is a severe complication causing repea… | The Knee 2026-08-13 15:01:34 | Source |
| Primary Soft Tissue Sarcomas of the Extremities: A Nationwide Retrosp… Limb salvage · PMID 42588730 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source : The aim of this study was to determine the oncologic outcomes for adult patients with primary extremity soft tissue s… | Cancers 2026-08-13 15:01:34 | Source |
| Risk of glaucoma among patients using tadalafil for lower urinary tra… Tadalafil · PMID 42552102 | Myopathies | Traitement symptomatique | Publication | PubMed | Voir la publication source To evaluate the association between tadalafil use for lower urinary tract symptoms (LUTS) and the risk of developing gl… | The British journal of ophthalmology 2026-08-13 15:01:34 | Source |
| Multidimensional Factors Influencing the Implementation of Early Mobi… The Impact of Early Mobilization Protocol in Patients in the ICU · PMID 42504084 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source This meta-synthesis of qualitative studies aimed to explore the experiences and perspectives of healthcare providers in… | Journal of advanced nursing 2026-08-13 15:01:28 | Source |
| Stepping it Up on Temporary Mechanical Circulatory Support - Mobiliza… The Impact of Early Mobilization Protocol in Patients in the ICU · PMID 42521141 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Early mobilization and prehabilitation in patients supported with temporary mechanical circulatory support (tMCS) have… | Journal of cardiac failure 2026-08-13 15:01:28 | Source |
| Clinical Impact of Complete Revascularization Before Left Ventricular… Revascularization · PMID 42590810 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Limited information exists regarding whether coronary revascularization by percutaneous coronary intervention (PCI) pri… | Artificial organs 2026-08-13 15:01:25 | Source |
| Postoperative perfusion volume on territorial arterial spin labeling… Revascularization · PMID 42591086 | Myopathies | Traitement symptomatique · Thérapie génique | Publication | PubMed | Voir la publication source Cerebral hyperperfusion syndrome (CHS) after revascularization for moyamoya disease (MMD) represents a failure of cereb… | Frontiers in physiology 2026-08-13 15:01:25 | Source |
| Cohort, gender and spatial patterns of delayed school enrolment in th… COVID-19 Pandemic and Parents of Disabled Children · PMID 42527426 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Delayed school enrolment can be an important marker for developmental delays and be associated with adverse health even… | Scientific reports 2026-08-13 15:01:22 | Source |
| School Readiness, Learning Challenges, and Lifestyle Changes Among Ch… COVID-19 Pandemic and Parents of Disabled Children · PMID 42532839 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source The COVID-19 pandemic disrupted schooling worldwide. While there is evidence of declines in academic performance, the l… | The Journal of school health 2026-08-13 15:01:22 | Source |
| From trunk and hip muscle function to class allocation: understanding… Manual muscular test · PMID 42539762 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Functional assessment in wheelchair basketball (WB) has three evaluation stages: analysis of isolated functions, combin… | Frontiers in sports and active living 2026-08-13 15:01:21 | Source |
| Precision and Error Propagation in Static MEMS-IMU Inertial Navigatio… test of walk · PMID 42590462 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source This paper investigates the precision and stochastic error propagation of navigation solutions obtained from a low-cost… | Sensors (Basel, Switzerland) 2026-08-13 15:01:20 | Source |
| The Effect of Whole-Body Cryotherapy on Exercise Capacity of Healthy… test of walk · PMID 42590172 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source : To analyze the effect of whole-body cryotherapy on exercise tolerance in healthy, training men related to their profe… | Journal of clinical medicine 2026-08-13 15:01:20 | Source |
| A qualitative study on the vulnerability factors of unintentional inj… Awareness Levels of Caregivers of Disabled Children · PMID 42577259 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Unintentional injuries represent a primary cause of child mortality worldwide. However, child injury research in China… | Frontiers in public health 2026-08-13 15:01:19 | Source |
| Intrathecal Baclofen Pump Therapy for Medically Refractory Stiff Pers… baclofen · PMID 42578902 | Myopathies | Immunomodulation · Traitement symptomatique | Publication | PubMed | Voir la publication source This study aimed to describe clinical outcomes, dosing, and safety of intrathecal baclofen (ITB) therapy in patients wi… | Neuromodulation : journal of the International Neuromo… 2026-08-13 15:01:12 | Source |
| On-Demand Hospital Manufacturing of Baclofen Lozenges Using Pharmaceu… baclofen · PMID 42581260 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Dysphagia in neurological patients complicates oral drug administration. Manual preparation of lozenges/troches lacks p… | Pharmaceutical research 2026-08-13 15:01:12 | Source |
| Cytokine Release Syndrome Induced by Neoadjuvant Pembrolizumab in Ear… Prednisolone · PMID 42583655 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Cytokine release syndrome (CRS) is a rare but potentially severe immune-related adverse event associated with immune ch… | Case reports in oncology 2026-08-13 15:00:21 | 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 |