Muscular dystrophy (MD) refers to a group of more than 30 genetic diseases characterized by progressive weakness and degeneration of the skeletal muscles that control movement. Some forms of MD are se...
Comprehensive, easy-to-understand information about this condition
How we create this content →Research studies investigating treatments and therapies for this condition.
Active Trials
Total Trials
Data from ClinicalTrials.gov Feb 10, 2026
Research studies investigating treatments and therapies for this condition.
Active Trials
Total Trials
Data from ClinicalTrials.gov Feb 10, 2026
The documentation surrounding muscular dystrophy is limited primarily due to the vast number of subtypes and the complexity of their genetic underpinnings. Because MD affects a relatively small population and the symptoms can vary greatly among individuals, systematic clinical studies have been challenging to conduct. Additionally, the lack of identified genes for many forms of MD further complicates the understanding and documentation of this condition.
To navigate your journey with muscular dystrophy, consider seeking a neurologist who specializes in neuromuscular disorders. This specialist can help tailor management strategies to your specific needs. Additionally, connecting with patient organizations such as the Muscular Dystrophy Association (http://mdausa.org) can provide valuable resources and support networks. You might also explore opportunities for participation in clinical trials, which can offer access to cutting-edge research and therapies. Lastly, while genetic counseling may not currently be applicable due to the lack of identified genes, it can be beneficial for understanding familial implications.
There are currently 254 active clinical trials exploring various aspects of muscular dystrophy, which may offer hope for future treatments. While there are no orphan drugs specifically designated for MD, the ongoing research indicates a commitment to understanding and addressing this condition. For more details on active trials, you can visit ClinicalTrials.gov and search for muscular dystrophy: https://clinicaltrials.gov/search?cond=muscular%20dystrophy.
Actionable guidance for navigating care for muscular dystrophy
To navigate your journey with muscular dystrophy, consider seeking a neurologist who specializes in neuromuscular disorders. This specialist can help tailor management strategies to your specific needs. Additionally, connecting with patient organizations such as the Muscular Dystrophy Association (http://mdausa.org) can provide valuable resources and support networks. You might also explore opportunities for participation in clinical trials, which can offer access to cutting-edge research and therapies. Lastly, while genetic counseling may not currently be applicable due to the lack of identified genes, it can be beneficial for understanding familial implications.
Consider asking your healthcare providers these condition-specific questions
Connect with organizations supporting the muscular dystrophy community
Helpful links for rare disease information and support
Clinical profile data for this condition is not yet available. Phenotype information may still be loading below.
Research studies investigating treatments and therapies for this condition.
Active Trials
Total Trials
Data from ClinicalTrials.gov Feb 10, 2026
Patient Advocacy Groups (PAGs) provide support, resources, and community for patients and caregivers.
Consider asking your healthcare providers these condition-specific questions
European rare disease database
Genetic and Rare Diseases Info Center
2 peer-reviewed sources from PubMed
AI-Generated Content: This summary was generated using AI. Content has been fact-checked. Always consult with qualified healthcare providers for medical guidance.
Organizations with approved therapies for this disease
No approved therapies yet
Research is ongoing — 1 company has orphan drug designations for this disease
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 muscular dystrophy
Updated Jan 26, 2026
The Muscular Dystrophy Association (MDA) is celebrating Rare Disease Day on February 28 by sharing community stories that highlight advancements in research, care, and advocacy for individuals with rare neuromuscular diseases. This initiative aims to enhance awareness and engagement among those affected by muscular dystrophy, ALS, and related conditions.