"Duchenne Muscular Dystrophy Multidisciplinary Management Guide (2025 Edition)" Release
Recently, the International Association for Neuromuscular Diseases (INMD) and experts from many countries around the world jointly released the "Guidelines for the Management of Multidisciplinary Duchenne Muscular Dystrophy (2025 Edition)". Based on the latest clinical research evidence, the guideline provides systematic recommendations for the diagnosis, treatment and long-term management of Duchenne muscular dystrophy (DMD), aiming to provide more comprehensive guidance to clinicians, patients and families. The following are the core content and hot topics of the guide that have been hotly discussed across the Internet in the past 10 days.
1. Key points for the core update of the guide
The 2025 version of the guide has been comprehensively revised based on the 2019 version, focusing on the following areas:
| field | Update content | Level of evidence |
|---|---|---|
| Early diagnosis | New standardized process for genetic testing | Class A |
| Drug treatment | Recommended hormone therapy combined with novel gene therapy | Level B |
| Rehabilitation management | Determine the intensity and frequency of exercise intervention | C-level |
| Heart monitoring | At least 2 cardiac function assessments per year | Class A |
2. Top 5 hot topics on the entire network
According to social media monitoring data, the discussion on DMD guidelines in the past 10 days has focused on the following topics:
| Ranking | topic | Discussion volume (10,000) |
|---|---|---|
| 1 | Possibility of gene therapy in medical insurance | 28.5 |
| 2 | Psychological support programs in home care | 19.2 |
| 3 | Application of new exoskeleton rehabilitation equipment | 15.7 |
| 4 | Construction of international diagnosis and treatment collaboration network | 12.3 |
| 5 | New standards for patient nutrition management | 9.8 |
3. Multidisciplinary management framework
Guidelines are first proposed"Four-dimensional management model", emphasizing the coordination of the following disciplines:
| Dimension | Responsible for the subject | Key interventions |
|---|---|---|
| Medical intervention | Neurology/Pediatrics/Cardiology | Drug management, complication prevention |
| Function maintenance | Rehabilitation/Orthopedics | Joint mobility training, orthotic adaptation |
| Social support | Psychology/social worker | Family consultation, educational resource docking |
| Scientific research transformation | Clinical Research Center | Participation in clinical trials and construction of biological sample bank |
4. Comparison of patient quality of life data
By comparing the data from 2015 to 2025, the guideline shows that standardized management significantly improves patient quality of life:
| index | 2015 | 2025 | Improvement range |
|---|---|---|---|
| Average survival age | 25.3 years old | 32.1 years old | +26.9% |
| Wheelchair time dependence | 10.2 years | 8.5 years | -16.7% |
| Emergency hospitalizations | 4.7 times/year | 2.3 times/year | -51.1% |
5. Expert consensus and disputes
During the development of the guidelines, the Expert Committee reached consensus and reservations on the following issues:
Consensus points:All DMD patients should start hormone therapy before the age of 3; a follow-up team including respiratory physicians must be established; gene therapy must be carried out in professional centers.
Controversy point:There are differences in the clinical positioning of stem cell therapy; the standards for use of nutritional supplements are not unified; transitional (puberty) management plans need further research.
6. Implementation suggestions and resource support
To promote the implementation of the guide, INMD has launched simultaneously:
1.Doctor Tool Kit:Including diagnostic flowcharts and drug dosage calculator
2.Patient Manual:12 language version of e-book
3.Training Program:Touring training will be carried out in 30 countries in 2025
The release of this guide marks a new stage of precision and standardization of DMD management, and its multidisciplinary collaboration model also provides important reference for the management of other rare diseases.
en
