Duchenne muscular dystrophy (DMD) is a neuromuscular disease characterized by rapidly progressive muscle weakness and wasting due to degeneration of skeletal and cardiac muscle.
Comprehensive, easy-to-understand information about this condition
How we create this content →Parent Project Muscular Dystrophy's mission is to end Duchenne muscular dystrophy.
Patient Advocacy Groups (PAGs) provide support, resources, and community for patients and caregivers.
Online Mendelian Inheritance in Man
European rare disease database
Genetic and Rare Diseases Info Center
Parent Project Muscular Dystrophy's mission is to end Duchenne muscular dystrophy.
Patient Advocacy Groups (PAGs) provide support, resources, and community for patients and caregivers.
Online Mendelian Inheritance in Man
European rare disease database
Genetic and Rare Diseases Info Center
Actionable guidance for navigating care for Duchenne muscular dystrophy
To effectively navigate care for Duchenne muscular dystrophy (DMD), consider seeking specialists such as a pediatric neurologist with neuromuscular expertise, a cardiologist for cardiac monitoring, and a pulmonologist for respiratory health. You may also benefit from physical and occupational therapy. Connect with patient organizations like Parent Project Muscular Dystrophy (https://www.parentprojectmd.org) and Cureduchenne (https://cureduchenne.org) for resources and support. Additionally, explore the Duchenne Registry (https://duchenneregistry.org) for participation in research. For genetic counseling, visit findageneticcounselor.com to locate a qualified professional who can assist in understanding the implications of the DMD gene variant for your family.
Consider asking your healthcare providers these condition-specific questions
Connect with organizations supporting the Duchenne muscular dystrophy community
Helpful links for rare disease information and support
Inheritance patterns describe how genetic conditions are passed from parents to children.
Research studies investigating treatments and therapies for this condition.
Active Trials
Total Trials
Data from ClinicalTrials.gov Feb 1, 2026
Parent Project Muscular Dystrophy's mission is to end Duchenne muscular dystrophy.
Patient Advocacy Groups (PAGs) provide support, resources, and community for patients and caregivers.
Online Mendelian Inheritance in Man
European rare disease database
Genetic and Rare Diseases Info Center
5 peer-reviewed sources from PubMed
AI-Generated Content: This summary was generated using AI. Always consult with qualified healthcare providers for medical guidance.
Kisho delivers this disease record via API, including phenotypes (HPO), genes, orphan drug designations, screening status, and PAG mapping, with version history and governance.
AI-curated news mentioning Duchenne muscular dystrophy
Updated Jan 30, 2026
The 23rd International Conference on Duchenne and Becker Muscular Dystrophy recently took place, focusing on advancements and challenges in the field. This event highlights ongoing efforts to improve awareness and support for affected individuals and families.
Sarepta Therapeutics reports that its gene therapy Elevidys for Duchenne muscular dystrophy shows continued benefits three years post-treatment, despite facing regulatory challenges and a significant workforce reduction. The therapy's efficacy comes amid scrutiny following patient deaths linked to liver injury, highlighting the risks associated with AAV therapies.
Regenxbio's RGX-202 gene therapy for Duchenne muscular dystrophy (DMD) demonstrated significant improvements in patient outcomes, exceeding expected disease progression at 12 and 18 months post-treatment in a pivotal trial. The company plans to submit a Biologics License Application (BLA) in mid-2026 via an accelerated approval pathway.
The HHS Secretary has accepted the recommendation to add Duchenne Muscular Dystrophy (DMD) to the Recommended Uniform Screening Panel (RUSP), following public comments and evidence-based reports. This decision could enhance early detection and intervention for DMD, impacting patient outcomes significantly.
NORD commends HHS for including metachromatic leukodystrophy (MLD) and Duchenne muscular dystrophy (DMD) in the Recommended Uniform Screening Panel, enhancing early detection for these rare diseases. This decision supports better patient outcomes through timely diagnosis.