Duchenne muscular dystrophy (DMD) is a severe X-linked genetic disease caused by mutations in the dystrophin gene, leading to progressive muscle breakdown that typically confines boys to wheelchairs by their early teens and affects the heart and breathing muscles over time. It is the most common and serious form of muscular dystrophy in children.
What's actually going on in research
Several exon-skipping drugs have been granted accelerated or conditional approval to restore partial dystrophin production in specific mutation subsets, and a gene therapy — delandistrogene moxeparvovec — has received approval in the US for younger children. Trials are testing more potent exon-skipping drugs, CRISPR-based gene correction, utrophin upregulators, and anti-inflammatory or muscle-preserving agents to complement or extend the reach of these advances.
Gene replacement therapy
Delandistrogene moxeparvovec delivers a shortened but functional micro-dystrophin gene via an AAV vector and is approved for ambulatory children under five; trials are extending this to older and non-ambulatory patients.
Exon-skipping drugs
Antisense oligonucleotides that skip specific exons to restore the dystrophin reading frame are approved for some mutations, and next-generation peptide-conjugated versions with improved muscle delivery are in active trials.
CRISPR gene correction
Early-phase trials are testing CRISPR-based approaches to permanently correct dystrophin mutations in muscle and cardiac cells, aiming for a one-time treatment that restores more complete protein function.
What to know before you search
Eligibility depends on the specific dystrophin mutation, age, ambulatory status, and prior corticosteroid or gene therapy use.
What types of trials are currently open
- Gene therapy trials — Testing AAV-delivered micro-dystrophin constructs across different age groups and mutation types.
- Exon-skipping trials — Evaluating antisense drugs and next-generation conjugates that restore dystrophin reading frame for specific mutations.
- CRISPR trials — Early-phase studies of gene-editing approaches to permanently correct the dystrophin gene.
- Cardioprotection trials — Studying drugs to slow or prevent the cardiomyopathy that develops in most DMD patients.
- Anti-inflammatory trials — Testing corticosteroid alternatives and other anti-inflammatory drugs to slow muscle degeneration.
Recently added Duchenne Muscular Dystrophy trials
DMD Gene Variants and Cardiac Dysfunction in Young Males With Dystrophinopathies
The goal of this observational study is to investigate whether the type, location, and extent of pathogenic variants in the DMD gene are associated with cardiac dysfunction in male children, adolescents, and young adults with dystrophinopathies. The study also evaluates whether cardiac biomarkers and electrocardiographic findings can facilitate the early identification of cardiac involvement. Participants will undergo electrocardiography, blood sampling for cardiac biomarker assessment, and transthoracic echocardiography, with cardiac dysfunction evaluated using ejection fraction (EF) and global longitudinal strain (GLS).
Invasive Home Ventilation in Denmark
The aim of this study is to describe national trends over the past 10 years in patients receiving invasive home mechanical ventilation (HMV) in Denmark. This includes indications for invasive HMV, diagnostic groups, and one-year mortality.
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