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 |
|---|---|---|---|---|---|---|---|
| Brain signatures of nociplastic pain: Fibromyalgia Index and descendi… Musculoskeletal Nociceptive Pain in Participants With Neuromuscular Disorders · PMID 40819274 | Myopathies | Traitement symptomatique | Publication | PubMed | Voir la publication source Nociplastic pain is defined by altered nociceptive processing in the absence of clear peripheral damage or somatosensor… | Brain : a journal of neurology 2026-08-16 13:25:16 | Source |
| Neuropathic and Nociplastic Pain Profiles are Common in Adult Chronic… Musculoskeletal Nociceptive Pain in Participants With Neuromuscular Disorders · PMID 38627292 | Myopathies | Neuroprotection · Immunomodulation · Traitement symptomatique | Publication | PubMed | Voir la publication source Chronic nonbacterial osteitis (CNO) is a rare musculoskeletal disease causing chronic bone pain. It is known that chron… | Calcified tissue international 2026-08-16 13:25:16 | Source |
| Placebo-controlled Phase 2 Trial of Drisapersen for Duchenne Muscular… Drisapersen · PMID 30128316 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source This double-blind, randomized, placebo-controlled Phase 2 study (NCT01462292) assessed the 24-week efficacy, safety, to… | Annals of clinical and translational neurology 2026-08-16 13:25:16 | Source |
| Benefits of Telemonitoring of Pulmonary Function-3-Month Follow-Up of… AioCare spirometer · PMID 35160307 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source In patients with Duchenne Muscular Dystrophy (DMD), the respiratory system determines the quality and length of life; t… | Journal of clinical medicine 2026-08-16 13:25:15 | Source |
| Baseline neurochemical excitability and individual differences in mot… Non-invasive Neuromodulation · PMID 42263535 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Motor skill acquisition arises from complex interactions between inhibitory and excitatory neurotransmission in sensori… | Cortex; a journal devoted to the study of the nervous… 2026-08-16 13:25:08 | Source |
| Protocol for simultaneous non-invasive electrocardiogram and respirat… Kinesiology tape · PMID 42455681 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Here, we present a protocol for non-invasive simultaneous recording of electrocardiogram (ECG) and breathing in conscio… | STAR protocols 2026-08-16 13:25:08 | Source |
| Prospective Study of Video Hand Opening Time as a Quantitative Measur… SOUL-DM1 — Symptoms and Outcome Measures for Upper- Limb Function in Myotonic Dystrophy Type 1… | Myopathies | Ralentissement de la progression · Traitement symptomatique · Thérapie génique | Publication | PubMed | Voir la publication source Delayed grip relaxation is a common symptom of myotonic dystrophy type 1 (DM1), differing from other muscle diseases. P… | Neurology 2026-08-16 13:25:04 | Source |
| Case Report: Overlap of long QT syndrome and catecholaminergic polymo… Mexiletine · PMID 42558477 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Calmodulinopathy is a rare -related hereditary channelopathy presenting with long QT syndrome (LQTS), less commonly wit… | Frontiers in pediatrics 2026-08-16 13:25:02 | Source |
| HIF-Prolyl Hydroxylase Inhibitor Desidustat Increases Pyruvate Kinase… GSK1278863 · PMID 40192567 | Myopathies | Neuroprotection · Traitement symptomatique | Publication | PubMed | Voir la publication source Sickle cell anemia and beta-thalassemia are the major hemoglobinopathies associated with anemia. Bone marrow transplant… | Drug development research 2026-08-16 13:24:56 | Source |
| An investigation of a novel public health campaign to improve underst… Pain Science Education · PMID 42176372 | Myopathies | Traitement symptomatique · Thérapie génique | Publication | PubMed | Voir la publication source Persistent pain is a high priority global public health issue. Misconceptions about persistent pain prevail in society… | Musculoskeletal science & practice 2026-08-16 13:24:52 | Source |
| Complex intervention including pain science education and patient-led… Pain Science Education · PMID 39739224 | Myopathies | Ralentissement de la progression · Traitement symptomatique | Publication | PubMed | Voir la publication source Up to 74% of breast cancer survivors (BCS) treated with aromatase inhibitor (AI) experience AI-induced musculoskeletal… | Journal of cancer survivorship : research and practice 2026-08-16 13:24:52 | Source |
| Diffusion-weighted magnetic resonance imaging as a promising techniqu… Magnetic Resonance Imaging and Ultrasound Comparison With Load Evaluation · PMID 42599582 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source A combination of morphological and functional imaging plays a crucial role for neuroendocrine tumors (NETs) management.… | Journal of endocrinological investigation 2026-08-16 13:24:52 | Source |
| Quantitative susceptibility mapping of the brain is associated with i… Magnetic Resonance Imaging and Ultrasound Comparison With Load Evaluation · PMID 41656561 | Myopathies | Immunomodulation | Publication | PubMed | Voir la publication source Accumulation of paramagnetic substances in brain tissue may constitute a feature of Alzheimer's disease (AD) associated… | Journal of cerebral blood flow and metabolism : offici… 2026-08-16 13:24:52 | Source |
| A Step-Up Underwater Endoscopic Mucosal Resection Strategy with Rescu… GenPHSat injection (Safety) · PMID 42111926 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Underwater endoscopic mucosal resection (UEMR) is an established treatment for non-pedunculated colorectal lesions, but… | DEN open 2026-08-16 13:24:48 | Source |
| Efficiency and Safety of Endoscopic Injection Sclerotherapy With Liga… GenPHSat injection (Safety) · PMID 42088735 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Endoscopic injection sclerotherapy with ligation (EISL) is a combined endoscopic treatment that involves endoscopic inj… | DEN open 2026-08-16 13:24:48 | Source |
| Real-time NMR analysis of glutamine metabolism for screening anti-can… Glutamine · PMID 42518517 | Myopathies | Ralentissement de la progression | Publication | PubMed | Voir la publication source Metabolic reprogramming is a substantial obstacle for anticancer drug screening, as targeted therapeutics often lose ef… | Magnetic resonance letters 2026-08-16 13:24:46 | Source |
| Transglutaminase 2 regulates innate immunity: mechanisms and therapeu… Glutamine · PMID 42053427 | Myopathies | Immunomodulation | Publication | PubMed | Voir la publication source Innate immunity constitutes the primary barrier against invading pathogens and plays a crucial role in coordinating the… | Oncoimmunology 2026-08-16 13:24:46 | Source |
| Creatine monohydrate for lean mass, strength, and bone density in pos… Creatine Monohydrate · PMID 42141930 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Menopause is accompanied by accelerated losses in muscle mass and strength and declining bone density. Whether creatine… | Journal of the International Society of Sports Nutriti… 2026-08-16 13:24:45 | Source |
| The emerging and evolving evidence supporting creatine as an ergogeni… Creatine Monohydrate · PMID 41870601 | Myopathies | Neuroprotection · Immunomodulation | Publication | PubMed | Voir la publication source Creatine is one of the most extensively studied ergogenic aids, with over three decades of research supporting its role… | Journal of the International Society of Sports Nutriti… 2026-08-16 13:24:45 | Source |
| Beyond rodents: A systematic review of antidepressant-like effects in… Desipramine · PMID 42538543 | Myopathies | Thérapie génique | Publication | PubMed | Voir la publication source The development of antidepressants has traditionally relied on vertebrate models. offers a promising alternative approa… | Alternatives to laboratory animals : ATLA 2026-08-16 13:24:34 | 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 |