Limb-Girdle Muscular Dystrophy Clinical Trials 2026 — Find Recruiting Studies

About Limb-Girdle Muscular Dystrophy

Limb-Girdle Muscular Dystrophies are a clinically and genetically heterogeneous group of disorders characterised by progressive weakness predominantly affecting the shoulder and pelvic girdle muscles. Over 30 genetic subtypes have been defined, with LGMD R1 (calpain-3 deficiency) and LGMD R2 (dysferlin deficiency) being the most prevalent. Cardiac and respiratory involvement varies significantly by subtype.

Common Clinical Features

Progressive proximal shoulder and hip girdle weakness Difficulty rising from the floor or climbing stairs Markedly elevated serum creatine kinase Scapular winging Cardiomyopathy or arrhythmia (subtype-dependent) Respiratory muscle weakness in advanced disease Calf hypertrophy in some subtypes

Clinical Trial Eligibility Tips

What to know before applying to Limb-Girdle Muscular Dystrophy trials.

Trials are highly subtype-specific; a next-generation sequencing (NGS) muscle gene panel or whole exome sequencing confirming your exact LGMD subtype is essential before searching

Protein immunohistochemistry from a muscle biopsy (e.g. calpain-3, dysferlin staining) complements genetic data and is often required for enrolment

North Star Ambulatory Assessment (NSAA) or Motor Function Measure (MFM) scores are standard baseline metrics — obtain these from a neuromuscular physiotherapist