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 |
|---|---|---|---|---|---|---|---|
| Blunt Tibial Injury Is Associated With Primary Amputation for Lower E… Primary major amputation · PMID 42107181 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Single-center civilian series report up to a 20% primary amputation rate following lower-extremity vascular trauma (LEV… | The Journal of surgical research 2026-08-16 13:24:05 | Source |
| Contemporary Outcomes of Staged Versus Primary Major Amputation for S… Primary major amputation · PMID 42349641 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Lower extremity amputation is a necessary, life-saving intervention for patients with severe foot infection. Previous s… | Annals of vascular surgery 2026-08-16 13:24:04 | Source |
| Dengzhanxixin injection for acute ischemic stroke after reperfusion t… Revascularization · PMID 42518968 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Revascularization for acute ischemic stroke (AIS) can salvage at-risk brain tissue, but reperfusion injury may reduce t… | IBRO neuroscience reports 2026-08-16 13:24:04 | Source |
| Host-state biomarkers and clinical factors for stratification and opt… Revascularization · PMID 42571258 | Myopathies | Immunomodulation · Thérapie cellulaire | Publication | PubMed | Voir la publication source Chronic limb-threatening ischemia (CLTI) is a severe manifestation of peripheral arterial disease associated with high… | Regenerative therapy 2026-08-16 13:24:04 | Source |
| Long-term safety and effectiveness of hybrid coronary revascularizati… Revascularization · PMID 41675882 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Hybrid coronary revascularization (HCR), a combination of percutaneous coronary intervention and coronary artery bypass… | Annals of medicine and surgery (2012) 2026-08-16 13:24:04 | Source |
| Re: "Contemporary Outcomes of Staged Versus Primary Major Amputation… Primary major amputation · PMID 42401234 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Résumé non disponible dans la base locale. | Annals of vascular surgery 2026-08-16 13:24:04 | Source |
| Feasibility and Effectiveness of the WHO Caregiver Skills Training Pr… COVID-19 Pandemic and Parents of Disabled Children · PMID 42525203 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Parent-mediated interventions are a core component of services for children with autism and other neurodevelopmental co… | Journal of autism and developmental disorders 2026-08-16 13:23:58 | Source |
| Biomechanics of manual wheeled propulsion in children and adolescents… Manual muscular test · PMID 42034074 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Manual wheelchair use supports independence and participation for children with neuromuscular disorders; however, pedia… | Clinical biomechanics (Bristol, Avon) 2026-08-16 13:23:57 | Source |
| A robotic perturbation trainer for transverse-plane gait perturbation… test of walk · PMID 42502775 | Myopathies | Traitement symptomatique · Thérapie génique | Publication | PubMed | Voir la publication source This study describes the development and implementation of a robotic perturbation-based training methodology for childr… | MethodsX 2026-08-16 13:23:56 | Source |
| Nigral biochemical and structural alterations following subacute expo… test of walk · PMID 42502326 | Myopathies | Remyélinisation indirecte / réparation · Neuroprotection · Immunomodulation | Publication | PubMed | Voir la publication source The substantia nigra is the critical part of the midbrain responsible for movement-related functions. Nigral alteration… | Toxicology reports 2026-08-16 13:23:56 | Source |
| A qualitative exploration of self-compassion for caregivers of childr… Awareness Levels of Caregivers of Disabled Children · PMID 40906039 | Myopathies | Ralentissement de la progression | Publication | PubMed | Voir la publication source Caregivers of children with physical disabilities face unique stressors and experience high levels of anxiety, depressi… | Rehabilitation psychology 2026-08-16 13:23:55 | Source |
| Downbeat nystagmus: a practical approach based on a clinical case. baclofen · PMID 42594882 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Spontaneous downbeat nystagmus (DBN) is the most common form of acquired nystagmus, characterized by a slow upward ocul… | Arquivos de neuro-psiquiatria 2026-08-16 13:23:43 | Source |
| Nipocalimab and other FcRn blockers in neuromuscular disorders. Nipocalimab · PMID 42398839 | Myopathies | Immunomodulation | Publication | PubMed | Voir la publication source Neonatal Fc receptor (FcRn) inhibition represents a novel and targeted therapeutic approach for autoimmune neuromuscula… | Pharmacology & therapeutics 2026-08-16 13:23:02 | Source |
| Prednisolone modulates CD8⁺ and regulatory T-cell activity to dampen… Prednisolone · PMID 41807927 | Myopathies | Ralentissement de la progression | Publication | PubMed | Voir la publication source Immune checkpoint inhibitors (ICIs) have transformed the treatment of advanced melanoma, yet their efficacy is limited… | Oncoimmunology 2026-08-16 13:22:09 | Source |
| Shared genetics of IgA nephropathy and cardiometabolic diseases revea… Prednisolone · PMID 42535737 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Immunoglobulin A nephropathy (IgAN), the most common primary glomerular disease and a leading cause of end-stage kidney… | Renal failure 2026-08-16 13:22:09 | Source |
| Biological Response of ZX50 Mg Alloy: The Role of Surface Microstruct… Study of Families With Twins or Siblings Discordant for Rheumatic Disorders · PMID 42573495 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source The surface mechanical attrition treatment (SMAT) and subsequent controlled annealing were performed on a ZX50 (Mg5Zn0.… | ACS biomaterials science & engineering 2026-08-16 13:21:48 | Source |
| How much is sexual minority stress confounded with familial causes? A… Study of Families With Twins or Siblings Discordant for Rheumatic Disorders · PMID 42572393 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Compared with heterosexual persons, non-heterosexual persons have worse mental health. Sexual Minority Stress Theory (S… | Psychological medicine 2026-08-16 13:21:48 | Source |
| Vulnerability to bullying victimization: an investigation of genetic… Study of Families With Twins or Siblings Discordant for Rheumatic Disorders · PMID 41910150 | Myopathies | Thérapie génique | Publication | PubMed | Voir la publication source Bullying victimization is associated with serious short- and long-term consequences, including mental health problems.… | European journal of psychotraumatology 2026-08-16 13:21:48 | Source |
| Antibody levels and immune memory in children with kaposiform hemangi… Sirolimus · PMID 42154819 | Myopathies | Immunomodulation | Publication | PubMed | Voir la publication source This study evaluated the safety, immunogenicity, and post-vaccination immune responses of timely, sequential catch-up v… | Human vaccines & immunotherapeutics 2026-08-16 13:21:43 | Source |
| Efficacy and safety of TPO-RA combined with sirolimus in the treatmen… Sirolimus · PMID 41844533 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Relapsed immune thrombocytopenia (ITP) poses challenges in treatment. Thrombopoietin receptor agonists (TPO-RAs) are re… | Hematology (Amsterdam, Netherlands) 2026-08-16 13:21:43 | 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 |