חיפוש חופשי

מאמרים

Young Adults and Parents’ Coping With Duchenne/Becker Muscular Dystrophy: A Focus Group Study

Lessons learned from the first national population-based genetic carrier-screening program for Duchenne muscular dystrophy

מחקר תפקודי ריאות והערכת חומרת המחלה – מרפאה רב תחומית שניידר

מחקר מלווה בנושא הכשרת כלבי שירות לילדים ולאנשים עם מוגבלות פיזית

תחומים רפואיים

How much dystrophin is needed for survival and function

The 109 week treatment effects of viltolarsen

263rd ENMC International Workshop: Focus on female carriers of dystrophinopathy

Evaluation of rAAVrh74 gene therapy vector

Very Low Residual Dystrophin Quantity Is Associated with Milder Dystrophinopathy

Targeting gut dysbiosis against inflammation and impaired autophagy in DMD

The Role of Associations in Reducing the Emotional and Financial Impact on Parents Caring for Children with DMD

Genetic counseling for the dystrophinopathies—Practice resource of the National Society of Genetic Counselors

Phenotype predictions for exon deletions/duplications

The Relation Between Nutritional Intake and Weight in 4-18 Year Old Patients with DMD

Understanding anxiety experienced by young males with Duchenne muscular dystrophy

Moving Beyond the 2018 Minimum International Care Considerations for Osteoporosis Management in DMD

Fat embolism syndrome in DMD: Report on a novel case and systematic literature review

Cognitive function in DMD carriers: personal case series and literature review

Vamorolone: First Approval

Functional and Clinical Outcomes among Non-ambulatory Patients

Children on performing the NSAA at home for children with Duchenne

Antisense and Gene Therapy Options for Duchenne Muscular Dystrophy Arising from Mutations in the N-Terminal Hotspot

An Open Label Exploratory Clinical Trial Evaluating Safety and Tolerability of Once-Weekly Prednisone in Becker and Limb-Girdle Muscular Dystrophy

BMI-z scores of boys with Duchenne muscular dystrophy already begin to increase before losing ambulation: a longitudinal exploration of BMI, corticosteroids and caloric intake

Inhibition of nonsense-mediated mRNA decay may improve stop codon read-through therapy for Duchenne muscular dystrophy

Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial.

Growth pattern trajectories in boys with Duchenne muscular dystrophy

Interplay of disability, caregiver impact, and out-of-pocket expenditures in Duchenne muscular dystrophy: a cohort study

The Role of Taurine in Skeletal Muscle Functioning and Its Potential as a Supportive Treatment for Duchenne Muscular Dystrophy

Development of a novel startle response task in Duchenne muscular dystrophy

Muscle histological changes in a large cohort of patients affected with Becker muscular dystrophy

Measuring carer quality of life in Duchenne muscular dystrophy

Treating Duchenne Muscular Dystrophy: The Promise of Stem Cells, Artificial Intelligence, and Multi-Omics

Cardiorespiratory management of Duchenne muscular dystrophy: emerging therapies, neuromuscular genetics, and new clinical challenges

A Long-Term Study Evaluating the Effects of Nicorandil Treatment on Duchenne Muscular Dystrophy-Associated Cardiomyopathy in mdx Mice

Martial Arts Training for Boys with Duchenne Muscular Dystrophy

Real-world and natural history data for drug evaluation in Duchenne muscular dystrophy

Effect of Different Corticosteroid Dosing Regimens on Clinical Outcomes in Boys With Duchenne Muscular Dystrophy

CRISPR editing as a therapeutic strategy for Duchenne muscular dystrophy

Age at Diagnosis for Duchenne Muscular Dystrophy: Why we must do better