Duchenne muscular dystrophy
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Publications and studies (1745)
- Randomized researchiParticipants were divided into groups by lottery and compared with each other. This reduces the chance that a difference is due to something other than the treatment. Assessment of left ventricular dyssynchrony by speckle tracking echocardiography in children with duchenne muscular dystrophy. (2022/01/01) ♡
- Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Differential fates of introns in gene expression due to global alternative splicing. (2022/01/01) ♡
- Two novel RNA-binding proteins identification through computational prediction and experimental validation. (2022/01/01) ♡
- Implementation of Motor Function Measure score percentile curves - Predicting motor function loss in Duchenne muscular dystrophy. (2022/01/01) ♡
- Electrocardiographic prediction of late gadolinium enhancement on cardiac magnetic resonance in Becker muscular dystrophy. (2022/01/01) ♡
- Patient reported quality of life in limb girdle muscular dystrophy. (2022/01/01) ♡
- Clinical advances of RNA therapeutics for treatment of neurological and neuromuscular diseases. (2022/01/01) ♡
- Compartmental nutritional status and respiratory muscle function assessment in subjects with neuromuscular diseases. (2022/01/01) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. The Relation Between Nutritional Intake and Body Weight in 4-18 Year Old Patients with DMD: What could possibly be done to prevent weight gain? (2022/01/01) ♡
- Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Trabecular Bone Volume Is Reduced, With Deteriorated Microstructure, With Aging in a Rat Model of Duchenne Muscular Dystrophy. (2022/01/01) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD) (2022-12-28) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Neuropsychological Profiles of Children With Duchenne Muscular Dystrophy and Its Effects on Motor Functions (2022-12-22) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Tamoxifen in Duchenne Muscular Dystrophy (2022-12-20) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Schulze Muscular Dystrophy Ability Clinical Study (2022-12-05) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Fast Troponin as a Biomarker to Assess Exercise-induced Muscle Damage in Muscle Diseases (2022-10-27) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Study Determining the Frequency of Duchenne Muscular Dystrophy and Late-onset Pompe Disease (2022-10-24) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502) (2022-10-19) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy (2022-10-13) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Quality and Independence of Gait Classification Scale for DMD (QIGS-DMD) (2022-10-04) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Exploratory Study of NS-089/NCNP-02 in DMD (2022-09-29) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Bisoprolol in DMD Early Cardiomyopathy (2022-09-28) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Phase 1/2 Study in Boys With Duchenne Muscular Dystrophy (2022-09-23) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Effects of Parental Influence on Physical Activity Level and Participation in Children With Duchenne Muscular Dystrophy (2022-09-09) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Treatment of a Single Patient With CRD-TMH-001 (2022-09-01) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. E-monitoring of PULMonary Function in Patients With Duchenne Muscular Dystrophy at Home (2022-08-26) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Finding the Optimum Regimen for Duchenne Muscular Dystrophy (2022-08-12) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Examination of the Relationship Between Foot - Body Posture and Balance and Gait in Duchenne Muscular Dystrophy (2022-07-27) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Treatment Effect of Tamoxifen on Patients With DMD (2022-07-25) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Examination of Lower Urinary System Symptoms With Duchenne Muscular Dystrophy (2022-07-19) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 (Vesleteplirsen) in Patients With Duchenne Muscular Dystrophy (DMD) (2022-07-06) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study to Assess Safety, Tolerability, and PK of EDG-5506 in Healthy Volunteers and Becker Muscular Dystrophy Adults (2022-06-28) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy (2022-06-21) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Reliability and Validity of the Turkish Version of the Upper Limb Short Questionnaire in Duchenne Muscular Dystrophy (2022-06-09) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Validating Cardiac MRI Biomarkers and Genotype-Phenotype Correlations for DMD (2022-05-10) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren (2022-04-05) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) (2022-04-05) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Telerehabilitation in Duchenne Muscular Dystrophy (2022-03-31) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Biomarker for Duchenne Muscular Dystrophy (2022-03-24) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Safety and Biomarker Response to (+)-Epicatechin in Becker Muscular Dystrophy (2022-03-23) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Effects of Low-level Mechanical Vibration on Bone Density in Ambulant Children Affected by Duchenne Muscular Dystrophy (2022-03-16) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Development of a New Instrument to Evaluate Gait Characteristics of Individuals With Duchenne Muscular Dystrophy (2022-03-07) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Use of Dynamic Arm Supports to Promote Activities of Daily Living in Individuals With DMD (2022-02-18) ♡
- Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Dysregulated Autophagy and Mitophagy in a Mouse Model of Duchenne Muscular Dystrophy Remain Unchanged Following Heme Oxygenase-1 Knockout. (2021/12/31) ♡
- Retracted publicationiThis research has been withdrawn by science itself, for example due to a flaw in the method or unreliable data. Do not use it as support and do not discuss it as evidence. Can symptomatic nmDuchenne carriers benefit from treatment with ataluren? Results of 193-month follow-up. (2021/12/31) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Early treatment with Ataluren of a 2-year-old boy with nonsense mutation Duchenne dystrophy. (2021/12/31) ♡
- Morbidity, Clinical Course and Vaccination against SARS-CoV-2 Virus in Patients with Duchenne Muscular Dystrophy: A Patient Reported Survey. (2021/12/30) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. [Family genetic analysis of paternal Dystrophin gene mutations in a case of female Duchenne muscular dystrophy]. (2021/12/28) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Integrating Whole-Genome Sequencing in Clinical Genetics: A Novel Disruptive Structural Rearrangement Identified in the Dystrophin Gene (DMD). (2021/12/22) ♡
- TRPC3, but not TRPC1, as a good therapeutic target for standalone or complementary treatment of DMD. (2021/12/20) ♡
- Investigating the Potential for Sulforaphane to Attenuate Gastrointestinal Dysfunction in mdx Dystrophic Mice. (2021/12/20) ♡
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