Duchenne Muscular Dystrophy
Duchenne muscular dystrophy or DMD is the most common of the muscular dystrophies, affecting approximately 1 in every 3,500 newborn boys. It is caused by a fault in a gene called the dystrophin or DMD gene. A fault in this gene stops the body making a protein called dystrophin. This protein is important in muscle fibres, and its absence results in muscle weakness that gets worse over time because muscle cells break down and are gradually lost.
Because the dystrophin gene is on the X chromosome, Duchenne muscular dystrophy affects only boys. Girls have two X chromosomes, so if one of these is unaffected it can usually compensate for the faulty one, while boys have one X and one Y chromosome, so if their single copy of the dystrophin gene is faulty, they have the symptoms of DMD, while girls with one affected gene and one normal one usually won’t show symptoms but can be “carriers”. This means that the disease can be passed on in families – a mother who is a carrier has a 50:50 chance of having a son who is affected. But in up to about a third of cases, the mutation arises spontaneously in the boy.
- Dual Task in Duchenne Muscular Dystrophy
- Quality and Independence of Gait Classification Scale for DMD (QIGS-DMD)
- Investigation of the Validity and Reliability of the Kinesthetic and Visual Imagery Questionnaire in Children With Duchenne Muscular Dystrophy
- Natural History of Duchenne Muscular Dystrophy Cardiomyopathy (DMD-CMP)
- Cultural Adaptation, Validity, and Reliability of the Turkish Version of North Star Ambulatory Assessment
- A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy
- Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
- E-monitoring of PULMonary Function in Patients With Duchenne Muscular Dystrophy at Home"
- Treatment of a Single Patient With CRD-TMH-001
- Examination of Lower Urinary System Symptoms With Duchenne Muscular Dystrophy
- Postural and Anthropometric Properties of Foot and Ankle of Patients With DMD
- The Relationship Between Foot Posture and Performance and Ambulation in Patients With Duchenne Muscular Dystrophy
- Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy
- Once Weekly Infant Corticosteroid Trial for DMD
- Schulze Muscular Dystrophy Ability Clinical Study
- Determine the Safety and Dose of EN001 in Patients With Duchenne Muscular Dystrophy(DMD)
- Bicycle Ergometer Training in Duchenne Muscular Dystrophy
- Physical Therapy Treatment on Children and Adolescents With Neurological Pathologies
- Telerehabilitation in Duchenne Muscular Dystrophy
- Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy
The aim of this overview is to inform patients and parents about the different therapeutic approaches for Duchenne muscular dystrophy currently under investigation, to describe the advantages and disadvantages of each approach and to list the hurdles that have to be overcome before these approaches can be applied to patients.