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 |
|---|---|---|---|---|---|---|---|
| SGT-003 NCT06138639 · A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE) | Myopathies | Thérapie génique | Phase 1/2 | RECRUITING | This is a Phase 3, double-blind, placebo-controlled study with the primary… À vérifier | ClinicalTrials.gov 2026-08-16 19:04:32 | Détail |
| ASP2957 NCT07052929 · Study of ASP2957 in Male Participants With X-linked Myotubular Myopathy Who Need… | Myopathies | Traitement symptomatique · Thérapie génique | Phase 1/2 | RECRUITING | X-linked myotubular myopathy (XLMTM) is a rare and serious condition prese… À vérifier | ClinicalTrials.gov 2026-08-16 19:04:31 | Détail |
| CT103A NCT07752264 · A Phase I/II Study of Eque-cel in Subjects Withimmune-mediated Necrotizing Myopa… | Myopathies | Thérapie cellulaire | Phase 1/2 | NOT_YET_RECRUITING | CAR-T autologue anti-BCMA (CT103A / equecabtagene autoleucel) visant les c… À vérifier | ClinicalTrials.gov NCT04561557 ; Cell 2025 (Qin et al.… 2026-08-16 19:04:31 | Détail |
| NKX019 NCT06733935 · A Phase 1/2 Study of NKX019 in Subjects With Immune-Mediated Diseases (Ntrust-2) | Myopathies | À vérifier | Phase 1/2 | RECRUITING | Primary objective: Safety and tolerability of NKX019, administered after l… À vérifier | ClinicalTrials.gov 2026-08-16 19:04:31 | Détail |
| RGX-202 NCT05693142 · AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular D… | Myopathies | Thérapie génique | Phase 2/3 | ACTIVE_NOT_RECRUITING | RGX-202 is a gene therapy designed to deliver a transgene for a novel micr… À vérifier | ClinicalTrials.gov 2026-08-16 18:10:04 | Détail |
| GENOMIT — Global Registry and Natural History Study for Mitochondrial… NCT05554835 · NCT05554835 | Myopathies | Remyélinisation · Remyélinisation indirecte / réparation | À vérifier | RECRUITING | The main goal of the project is provision of a global registry for mitocho… À vérifier | ClinicalTrials.gov 2026-08-16 18:10:03 | Détail |
| AIM — Autoimmune Intervention Mastery Course Study NCT05057676 · NCT05057676 | Myopathies | Traitement symptomatique | Non applicable | ACTIVE_NOT_RECRUITING | The goal of this project is to critically evaluate the effectiveness of us… À vérifier | ClinicalTrials.gov 2026-08-16 18:10:02 | Détail |
| DYNE-251 NCT05524883 · DELIVER — Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic S… | Myopathies | À vérifier | Phase 1/2 | ACTIVE_NOT_RECRUITING | The primary purpose of this study is to evaluate the safety, tolerability,… À vérifier | ClinicalTrials.gov 2026-08-16 12:59:24 | Détail |
| ENTR-601-44 NCT07037862 · ELEVATE-44 — A Study in Participants With Duchenne Muscular Dystrophy Amenable t… | Myopathies | Thérapie génique | Phase 1/2 | RECRUITING | This is a study of the investigational medicine ENTR-601-44 in participant… À vérifier | ClinicalTrials.gov 2026-08-16 12:59:24 | Détail |
| ENTR-601-45 NCT07038824 · ELEVATE-45 — A Study in Participants With Duchenne Muscular Dystrophy Amenable t… | Myopathies | Thérapie génique | Phase 1/2 | RECRUITING | This is a study of the investigational medicine ENTR-601-45 in participant… À vérifier | ClinicalTrials.gov 2026-08-16 12:59:24 | Détail |
| Scapular Stabilization Exercises Combined With Thoracic Mobilization… NCT07766148 · NCT07766148 | Myopathies | Traitement symptomatique | Non applicable | COMPLETED | Upper-back pain is common among breastfeeding mothers and may be associate… À vérifier | ClinicalTrials.gov 2026-08-16 12:59:24 | Détail |
| SGT-001 NCT03368742 · IGNITE DMD — Microdystrophin Gene Transfer Study in Adolescents and Children Wit… | Myopathies | À vérifier | Phase 1/2 | ACTIVE_NOT_RECRUITING | This is a controlled, open-label, single-ascending dose study to evaluate… À vérifier | ClinicalTrials.gov 2026-08-16 12:59:24 | Détail |
| SRP-1001 for Injection NCT06131983 · Study of SRP-1001 in Adult and Adolescent Participants With Facioscapulohumeral… | Myopathies | À vérifier | Phase 1/2 | RECRUITING | The purpose of this study is to evaluate the safety, tolerability, pharmac… À vérifier | ClinicalTrials.gov 2026-08-16 12:59:24 | Détail |
| SRP-1003 IV Infusion NCT06138743 · Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy | Myopathies | À vérifier | Phase 1/2 | RECRUITING | This is a phase 1/2a double-blinded, placebo-controlled, dose-escalating s… À vérifier | ClinicalTrials.gov 2026-08-16 12:59:24 | Détail |
| Comparison of Eccentric Muscle Training and Proprioceptive Neuromuscu… NCT06407479 · NCT06407479 | Myopathies | Ralentissement de la progression · Traitement symptomatique | Non applicable | COMPLETED | This trial compared eccentric exercise training and the proprioceptive neu… À vérifier | ClinicalTrials.gov 2026-08-16 12:59:23 | Détail |
| Descartes-08 NCT07391605 · Descartes-08 in Autoantibody Myositis | Myopathies | À vérifier | Phase 2 | RECRUITING | This is a Phase IIb study to evaluate the safety and preliminary efficacy… À vérifier | ClinicalTrials.gov 2026-08-16 12:59:23 | Détail |
| FIBOBS — Fibromyalgia Syndrome in France: The Role of Assessment and… NCT04775368 · NCT04775368 | Myopathies | Traitement symptomatique | Non applicable | COMPLETED | The FIBOBS study is a multi-centered, observational, prospective study car… À vérifier | ClinicalTrials.gov 2026-08-16 12:59:23 | Détail |
| Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC) NCT06579352 · Study of UC-MSC in DMD Patients | Myopathies | Immunomodulation · Thérapie cellulaire | Phase 2 | ACTIVE_NOT_RECRUITING | The primary objective of this study is to provide UC-MSC treatment to pati… À vérifier | ClinicalTrials.gov 2026-08-16 12:59:23 | Détail |
| ISADER — Adduction and Infraspinatus Activity in Shoulder Pain NCT07765056 · NCT07765056 | Myopathies | Traitement symptomatique · Thérapie génique | Non applicable | COMPLETED | The goal of this randomized crossover trial is to determine whether differ… À vérifier | ClinicalTrials.gov 2026-08-16 12:59:23 | Détail |
| Prednisolone NCT05412394 · Once Weekly Infant Corticosteroid Trial for DMD | Myopathies | Immunomodulation · Thérapie génique | Phase 4 | RECRUITING | The goal of this clinical trial is to learn how different treatments affec… À vérifier | ClinicalTrials.gov 2026-08-16 12:59:23 | 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 |
|---|---|---|---|---|---|---|---|
| Tests of dynamic balance, motor function and fear of falling as indic… Fear of Falling in Muscular Dystrophy · PMID 40645042 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source This study aims to examine dynamic balance, motor function, and fear of falling (FOF) as indicators of fall risk in chi… | European journal of paediatric neurology : EJPN : offi… 2026-07-08 20:14:58 | Source |
| New insights into an old vaccine: potency and breadth of neutralizing… Yellow Fever vaccine (17D) · PMID 40766119 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source The yellow fever vaccine 17D has controlled yellow fever for almost 90 years and is considered highly successful. Howev… | medRxiv : the preprint server for health sciences 2026-07-08 20:14:57 | Source |
| New insights into an old vaccine: potency and breadth of neutralizing… Yellow Fever vaccine (17D) · PMID 41670061 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source The yellow fever vaccine 17D has controlled yellow fever for almost 90 years and is considered highly successful. Howev… | The Journal of infectious diseases 2026-07-08 20:14:57 | Source |
| Early mobilization in the neurotrauma intensive care unit: a service… The Impact of Early Mobilization Protocol in Patients in the ICU · PMID 42388707 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Early mobilization (EM) refers to active exercise commenced while patients are on the intensive care unit (ICU). Althou… | Frontiers in neurology 2026-07-08 20:14:56 | Source |
| Nursing-led stepwise pain-management protocol improves postoperative… The Impact of Early Mobilization Protocol in Patients in the ICU · PMID 42007103 | Myopathies | Traitement symptomatique | Publication | PubMed | Voir la publication source To assess the impact of a stepwise pain-management protocol on postoperative recovery, pain control, complication rate,… | American journal of translational research 2026-07-08 20:14:56 | Source |
| Experiences of women with disabilities accessing maternal and newborn… Awareness Levels of Caregivers of Disabled Children · PMID 42192434 | Myopathies | Ralentissement de la progression · Thérapie génique | Publication | PubMed | Voir la publication source Despite global commitments to inclusive and equitable maternal and newborn health (MNH) care, women with disabilities i… | Reproductive health 2026-07-08 20:14:50 | Source |
| The effect of a mindfulness-based stress reduction program on stress… Awareness Levels of Caregivers of Disabled Children · PMID 42297152 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Mothers of children with physical disabilities often experience caregiving-related stress that may negatively affect th… | Journal of affective disorders 2026-07-08 20:14:50 | Source |
| One-day rejuvenation: a preliminary case series of a multimodal proto… Botulinum toxin type A · PMID 42418230 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Same-day multimodal aesthetic treatments may optimize patient convenience, but concerns remain regarding possible therm… | Journal of cosmetic and laser therapy : official publi… 2026-07-08 20:14:49 | Source |
| Activation of GABAR alleviates colitis by reprogramming macrophage po… baclofen · PMID 42413737 | Myopathies | Immunomodulation | Publication | PubMed | Voir la publication source Although the metabotropic GABA type B receptor (GABAR) has emerged as an immune modulator, its specific role in regulat… | Biochemical pharmacology 2026-07-08 20:14:47 | Source |
| Long-Term Effects of Early Postnatal Administration of R-Baclofen on… baclofen · PMID 42411441 | Myopathies | Traitement symptomatique | Publication | PubMed | Voir la publication source Contactin-associated protein-like 2 () is a highly expressed gene during development, coding for the cell adhesion mole… | Journal of integrative neuroscience 2026-07-08 20:14:47 | Source |
| Intermittent Theta-Burst Stimulation and Depressive Symptoms in Major… Intermittent theta burst stimulation · PMID 42418203 | Myopathies | Traitement symptomatique | Publication | PubMed | Voir la publication source Intermittent theta-burst stimulation (iTBS) is an established treatment for major depressive disorder (MDD), but sham-c… | JAMA network open 2026-07-08 20:14:19 | Source |
| Intermittent Theta-Burst Stimulation for Depression-Delineating Speci… Intermittent theta burst stimulation · PMID 42418204 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Résumé non disponible dans la base locale. | JAMA network open 2026-07-08 20:14:19 | Source |
| Feasibility and behavioral impact of a wearable-supported digital hea… test of walk · PMID 42416388 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source This proof-of-concept study examined whether wearable devices integrated with the Welloop mobile application and a ligh… | Digital health 2026-07-08 15:43:04 | Source |
| Five-Year Outcomes of Paclitaxel-Coated Balloons in Patients with Dia… test of walk · PMID 42416524 | Myopathies | Thérapie génique | Publication | PubMed | Voir la publication source Long-term outcomes of paclitaxel-coated drug-coated balloons (DCBs) in patients with diabetes mellitus (DM) and infrain… | Vascular health and risk management 2026-07-08 15:43:04 | Source |
| Disparities in deworming coverage between children with and without d… COVID-19 Pandemic and Parents of Disabled Children · PMID 42264499 | Myopathies | Ralentissement de la progression | Publication | PubMed | Voir la publication source Children with disabilities experience lower school enrolment rates and may be systematically excluded from school-based… | BMJ global health 2026-07-08 12:06:58 | Source |
| Neurodevelopmental treatment needs after severe acute respiratory syn… COVID-19 Pandemic and Parents of Disabled Children · PMID 42050906 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source ObjectiveThis cohort study examined whether documented coronavirus disease 2019 in young children affects the need for… | The Journal of international medical research 2026-07-08 12:06:58 | Source |
| Aquatic training improves muscle strength and functional mobility in… Manual muscular test · PMID 42398272 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Myotonic dystrophy type 1 (DM1) is the most common adult muscular dystrophy and is characterized by progressive muscle… | Neuromuscular disorders : NMD 2026-07-08 12:06:57 | Source |
| Gaming Technology in Pediatric Cerebral Palsy and Related Neuromuscul… Manual muscular test · PMID 42206959 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source BackgroundChildren with cerebral palsy (CP) and related neuromuscular conditions often experience barriers to physical… | NeuroRehabilitation 2026-07-08 12:06:57 | Source |
| The reliability and validity of the 30-second chair sit-to-stand test… test of walk · PMID 42414666 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source This study investigated the reliability and validity of the five-repetition sit-to-stand (5xSTS) and 30-second chair si… | European spine journal : official publication of the E… 2026-07-08 12:06:56 | Source |
| Yiqi Fumai Lyophilized Injection for Improving Exercise Tolerance in… test of walk · PMID 42413004 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source High mortality and prevalence rates are hallmarks of chronic heart failure (CHF). Patients frequently have a much lower… | JMIR research protocols 2026-07-08 12:06:56 | 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 |