Limb-girdle muscular dystrophy (LGMD) is a heterogeneous group of muscular dystrophies characterized by proximal weakness affecting the pelvic and shoulder girdles. Cardiac and respiratory impairment ...
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 1, 2026
Research studies investigating treatments and therapies for this condition.
Active Trials
Total Trials
Data from ClinicalTrials.gov Feb 1, 2026
The documentation on limb-girdle muscular dystrophy is limited due to its heterogeneous nature and the fact that it affects a relatively small population, with a prevalence of 1-9 in 100,000. This rarity has resulted in fewer systematic clinical studies, making it difficult to establish comprehensive clinical features and genetic causes. Ongoing research aims to better characterize the condition and improve understanding.
To navigate limb-girdle muscular dystrophy, consider consulting a neurologist with expertise in neuromuscular disorders, particularly those specializing in muscular dystrophies. Engaging with the LGMD Awareness Foundation can provide valuable resources and support. Additionally, you may want to explore opportunities to participate in patient registries or natural history studies, which can contribute to a better understanding of the condition. Genetic counseling may also be beneficial, especially to discuss family implications and future planning.
There are currently 25 active clinical trials investigating various aspects of limb-girdle muscular dystrophy. Notable orphan drugs in development include Angiotensin (1-7), nandrolone, and prednisone. For more information on ongoing trials, you can visit ClinicalTrials.gov and search for limb-girdle muscular dystrophy. This research holds promise for future treatment options and improved management of the condition.
Actionable guidance for navigating care for limb-girdle muscular dystrophy
To navigate limb-girdle muscular dystrophy, consider consulting a neurologist with expertise in neuromuscular disorders, particularly those specializing in muscular dystrophies. Engaging with the LGMD Awareness Foundation can provide valuable resources and support. Additionally, you may want to explore opportunities to participate in patient registries or natural history studies, which can contribute to a better understanding of the condition. Genetic counseling may also be beneficial, especially to discuss family implications and future planning.
Consider asking your healthcare providers these condition-specific questions
Connect with organizations supporting the limb-girdle 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 1, 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
AI-Generated Content: This summary was generated using AI. Content has been fact-checked. 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.