Becker muscular dystrophy is a genetic disorder caused by mutations in the dystrophin gene, leading to progressive muscle weakness that typically begins in the legs and pelvis during childhood or adolescence. It affects about 1 in 18,000 to 30,000 males and follows a milder course than Duchenne muscular dystrophy. Treatment focuses on managing symptoms, preserving mobility, and monitoring heart and breathing function.
What's actually going on in research
Trials are testing gene therapies that deliver functional dystrophin genes, exon-skipping drugs that help cells make more usable dystrophin protein, and anti-inflammatory drugs that may slow muscle damage. Researchers are also studying heart-protective therapies, since cardiomyopathy is a leading concern, and ways to predict disease progression based on specific mutations.
Gene therapy
Several trials are delivering functional copies of micro-dystrophin genes using viral vectors. Early results show the body can produce measurable dystrophin protein after a single infusion.
Exon-skipping therapies
These drugs help cells skip over mutated sections of the dystrophin gene to produce a shorter but functional protein. Multiple exon-skipping compounds are in trials, targeting different mutations.
Cardiac therapies
Studies are testing whether early use of heart medications can prevent or delay cardiomyopathy. Researchers are also examining imaging markers that could identify heart problems before symptoms appear.
What to know before you search
Eligibility typically depends on confirmed dystrophin gene mutation, age, current muscle function, heart status, and whether the specific mutation matches the therapy being tested.
What types of trials are currently open
- Gene therapy trials — Testing one-time infusions of viral vectors carrying dystrophin genes to see if the body can produce the missing protein.
- Exon-skipping trials — Testing drugs that alter how cells read the dystrophin gene, aiming to produce more functional protein.
- Cardiac trials — Testing medications and interventions to protect heart function and prevent cardiomyopathy.
- Anti-inflammatory trials — Testing drugs that may reduce inflammation and slow muscle breakdown.
- Natural history studies — Following people with Becker muscular dystrophy over time to understand how the disease progresses and what factors predict outcomes.
Recently added Becker Muscular Dystrophy trials
Track pain and movement changes over 6 months
1\. Brief Summary This prospective observational study aims to evaluate the 6-month longitudinal changes in chronic pain, pain characteristics, coping strategies, and functional performance in children and adolescents aged 7 to 18 years diagnosed with Duchenne Muscular Dystrophy (DMD), with a primary focus on comparing ambulatory and non-ambulatory disease stages. Pediatric DMD patients followed at the University of Health Sciences Antalya Training and Research Hospital Neuromuscular Diseases Center will undergo comprehensive evaluations at baseline (Month 0) and at 6 months follow-up (Month 6). Pain parameters will be evaluated using the Adolescent Pediatric Pain Tool (APPT) to assess pain intensity, body localization, and sensory/affective/evaluative/temporal qualities, and the Brief Pain Inventory (BPI) 7-item Pain Interference Scale to determine pain interference with daily activities. Pain coping strategies will be assessed using the Pediatric Pain Coping Inventory (PPCI, adolescent and parent versions). Functional and motor assessments will include the North Star Ambulatory Assessment (NSAA), 6-Minute Walk Test (6MWT), stair climb/descend tests, Vignos and Brooke scales, handgrip dynamometry, manual muscle testing (MMT), and range of motion (ROM) and contracture evaluations, alongside anthropometrics and quality of life. The central hypotheses are that non-ambulatory patients experience chronic pain more frequently, with longer duration, greater intensity, and across a higher number of anatomical sites than ambulatory patients. Furthermore, the study hypothesizes that anatomical pain sites, pain-aggravating and pain-relieving factors, and pain coping mechanisms differ significantly between ambulatory and non-ambulatory stages.
Bone Health in Adults With Duchenne Muscular Dystrophy: A Survey on Osteoporosis, Fractures and Treatment.
Background and Rationale Duchenne muscular dystrophy (DMD) is a genetic disorder caused by mutations in the dystrophin gene, resulting in progressive muscle weakness and multiple complications, including respiratory and cardiac impairment, osteoporosis (OP), and fragility fractures (FF). Bone fragility is a major concern, as lower-limb fractures may accelerate the loss of independent ambulation and complicate daily care. Despite increased life expectancy, bone health in adults with DMD remains insufficiently investigated. This study aims to address this knowledge gap and improve the understanding and management of skeletal complications in this population. Objectives The study aims to: (1) determine the prevalence of fragility fractures in adults with DMD; (2) identify childhood and adolescent risk or protective factors affecting adult bone health; (3) collect data on fracture treatment; (4) measure serum analytes involved in bone metabolism and bone-muscle crosstalk; and (5) identify genetic modifiers associated with increased fracture risk. Study Population Approximately 200 patients with DMD aged over 18 years will be recruited from 15 Italian tertiary referral centers specializing in neuromuscular diseases. A control group of healthy males aged over 18 years will be recruited exclusively at the coordinating center. Controls will undergo only a blood collection of approximately 3 mL for serum analysis. Study Design and Procedures Patients will undergo respiratory assessment, including peak expiratory flow (PEF), forced vital capacity (FVC), and respiratory support requirements; orthopedic assessment of scoliosis, contractures, deformities, and previous spinal fusion surgery; motor function assessment using the North Star Ambulatory Assessment (NSAA); upper-limb function assessment using the Performance of the Upper Limb (PUL) scale; and the Six-Minute Walk Test (6MWT). Imaging procedures will include a lateral thoracolumbar spine radiograph (T4-L4) and dual-energy X-ray absorptiometry (DXA) of the lumbar spine (L1-L4) and total body less head (TBLH). Clinical and medical history data will be recorded using a dedicated case report form (CRF). Biological Samples and Analyses One blood sample of approximately 3 mL will be collected for serum extraction and analysis of metabolites involved in bone metabolism and bone-muscle crosstalk. Samples will be centralized at the coordinating center and then sent to the University of Campania "Luigi Vanvitelli" for analysis. Healthy control samples will be used for comparison. Two additional blood tubes, totaling approximately 6 mL, will be collected for DNA extraction and genome-wide association studies (GWAS), aimed at identifying genetic modifiers associated with increased fracture risk. These samples will be centralized and subsequently sent to the University of Padua for genetic analysis. Data Management and Ethics Study data will be anonymized and managed in accordance with the General Data Protection Regulation (GDPR, EU 2016/679) and the Declaration of Helsinki. Patients and/or their legally authorized representatives will receive the study information and provide written informed consent before participation. Investigators will declare any potential conflicts of interest. Expected Outcomes The study will characterize bone health and fragility fracture prevalence in adults with DMD, describe fracture management, and investigate clinical, developmental, biochemical, and genetic factors associated with fracture risk. The findings may help improve fracture prevention, bone health monitoring, and clinical management in this population.
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