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
Filtrer toute la vue
Chaque choix s’applique immédiatement aux quatre sections. Le filtre « type de SEP » n’apparaît que lorsque la sclérose en plaques est sélectionnée.
1012 résultats correspondants
· 46 visibles sur cette page
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 |
|---|---|---|---|---|---|---|---|
| Six approved drugs for alcohol use disorder: algorithms for their use… baclofen · PMID 42476146 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source The pharmacological management of alcohol use disorder (AUD) currently includes six medications approved by different r… | Alcohol and alcoholism (Oxford, Oxfordshire) 2026-07-22 21:22:33 | Source |
| Beyond intraocular pressure: vascular and metabolic modifiers of opti… Metformin · PMID 42484773 | Myopathies | Ralentissement de la progression · Thérapie génique | Publication | PubMed | Voir la publication source To critically integrate epidemiological, mechanistic, and clinical evidence linking systemic blood pressure dysregulati… | International ophthalmology 2026-07-22 21:21:06 | Source |
| Case Report: Early-onset euglycemic diabetic ketoacidosis following h… Metformin · PMID 42483676 | Myopathies | Traitement symptomatique | Publication | PubMed | Voir la publication source Sodium-glucose cotransporter 2 (SGLT2) inhibitors are associated with euglycemic diabetic ketoacidosis (euDKA), typical… | Frontiers in endocrinology 2026-07-22 21:21:06 | Source |
| Interim Safety Profile From the Feasibility Study of the BrainGate Ne… BrainGate Neural Interface System · PMID 36639237 | Myopathies | Ralentissement de la progression | Publication | PubMed | Voir la publication source Brain-computer interfaces (BCIs) are being developed to restore mobility, communication, and functional independence to… | Neurology 2026-07-18 15:48:46 | Source |
| Neural Point-and-Click Communication by a Person With Incomplete Lock… BrainGate Neural Interface System · PMID 25385765 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source A goal of brain-computer interface research is to develop fast and reliable means of communication for individuals with… | Neurorehabilitation and neural repair 2026-07-18 15:48:46 | Source |
| Multimodality Risk Stratification in Athletes With Long QT Syndrome. Mexiletine · PMID 42460964 | Myopathies | Thérapie génique | Publication | PubMed | Voir la publication source Long QT syndrome has traditionally led to exercise restriction due to concerns for exertion-triggered arrhythmia and su… | JACC. Case reports 2026-07-18 15:48:44 | Source |
| Efficacy of perifracture site injections for pain control following d… GenPHSat injection (Efficacy) · PMID 42469861 | Myopathies | Traitement symptomatique | Publication | PubMed | Voir la publication source Surgical site analgesic injections are used as part of multimodal analgesia in elective orthopedic surgery, but their e… | Journal of orthopaedic surgery and research 2026-07-18 15:48:08 | Source |
| Denecimig (Mim8) prophylaxis once-every-2-weeks for hemophilia A with… GenPHSat injection (Safety) · PMID 42469164 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Denecimig (Mim8) is a next-generation, activated factor VIII mimetic, fully human bispecific IgG4 antibody in developme… | Blood advances 2026-07-18 15:48:07 | Source |
| Long-term efficacy and safety of intra-articular corticosteroid injec… GenPHSat injection (Safety) · PMID 42470097 | Myopathies | Ralentissement de la progression · Traitement symptomatique · Thérapie génique | Publication | PubMed | Voir la publication source Knee osteoarthritis (KOA) is a highly prevalent condition worldwide, resulting in a significant symptom burden and dimi… | Expert opinion on drug safety 2026-07-18 15:48:07 | Source |
| Examining the feasibility and preliminary effects of resistance exerc… Creatine Monohydrate · PMID 42455839 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source The purpose of this trial was to assess the feasibility, acceptability and safety of a 10-week hybrid (in-clinic and vi… | PloS one 2026-07-18 15:48:05 | Source |
| A case report of Good syndrome: Diarrhea and dyspnea. IvIg · PMID 42470030 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Good syndrome (GS) is a rare acquired immunodeficiency disorder characterized by thymoma, hypogammaglobulinemia, and pe… | Medicine 2026-07-18 15:47:59 | Source |
| Knowledge graph visualization and bibliometric analysis of research o… IvIg · PMID 42470076 | Myopathies | Thérapie génique | Publication | PubMed | Voir la publication source Coronary artery lesions (CAL) complicating Kawasaki disease (KD) are a central concern affecting the long-term cardiova… | Medicine 2026-07-18 15:47:59 | Source |
| Evaluation of the "Los Filabres" Protocol on Behavioral and Psycholog… Behavioral Interventions for Controlling Oral Behaviors · PMID 42450944 | Myopathies | Traitement symptomatique | Publication | PubMed | Voir la publication source Behavioral and psychological symptoms of dementia (BPSD) are highly prevalent among nursing home residents and represen… | Healthcare (Basel, Switzerland) 2026-07-18 15:47:58 | Source |
| Linguistical and psychometric validation of the MIND Youth Questionna… Behavioral Interventions for Controlling Oral Behaviors · PMID 42449006 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source The Mind Youth Questionnaire is a multidimensional health-related quality-of-life questionnaire designed for pediatric… | Journal of patient-reported outcomes 2026-07-18 15:47:58 | Source |
| Adrenal rather than central dysfunction limits HPA axis recovery afte… Chronic Corticosteroid Therapy · PMID 42464759 | Myopathies | Thérapie génique | Publication | PubMed | Voir la publication source Glucocorticoid-induced adrenal insufficiency (GIAI) can persist for months after discontinuation of chronic corticoster… | Endocrinology 2026-07-18 15:47:55 | Source |
| Erectile dysfunction severity and penile Doppler findings in pelvic f… Tadalafil · PMID 42465782 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source Erectile dysfunction (ED) is a common and multifactorial complication after pelvic fracture urethral injury (PFUI), yet… | Indian journal of urology : IJU : journal of the Urolo… 2026-07-18 15:47:51 | Source |
| Targeting canonical TGFβ/SMAD3 and ERK1/2 signaling in human valve in… Tadalafil · PMID 42461450 | Myopathies | Ralentissement de la progression · Immunomodulation | Publication | PubMed | Voir la publication source Rheumatic heart disease (RHD) is a chronic sequel of acute rheumatic fever characterized by sustained inflammation, fib… | Journal of physiology and biochemistry 2026-07-18 15:47:51 | Source |
| Perceived Impacts of Caring for a Child With Disability on Siblings a… COVID-19 Pandemic and Parents of Disabled Children · PMID 42454384 | Myopathies | À vérifier | Publication | PubMed | Voir la publication source This study investigated the perceived implications of caring on siblings and parents under COVID-19 lockdown in Austral… | Physical & occupational therapy in pediatrics 2026-07-18 15:47:46 | Source |
| Comparison of the Effects of Telerehabilitation-Based Motor and Cogni… test of walk · PMID 42468927 | Myopathies | Thérapie génique | Publication | PubMed | Voir la publication source The co-occurrence of motor and cognitive impairments in Parkinson's disease underscores the importance of dual-task tra… | Journal of aging and physical activity 2026-07-18 15:47:44 | Source |
| Does combining walked distance and oxygenation obtained during the si… test of walk · PMID 42468472 | Myopathies | Thérapie génique | Publication | PubMed | Voir la publication source The six-minute walk distance (6MWD) is widely used to assess functional status and prognosis in interstitial lung disea… | Heart & lung : the journal of critical care 2026-07-18 15:47:44 | 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 |