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Updated: Jul 11, 2025

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Patterns of Clinical Progression Among Patients With Autosomal Recessive Limb-Girdle Muscular Dystrophy: A Systematic
Antoinette Cheung1, Ivana F Audhya2, Shelagh M Szabo1
1Broadstreet HEOR, Vancouver, BC, Canada.
Journal of Clinical Neuromuscular Disease
|November 14, 2023
Summary
Autosomal recessive limb-girdle muscular dystrophy (LGMDR) frequently leads to loss of ambulation (LOA). Cardiac and respiratory issues are common, particularly in specific LGMDR subtypes, impacting clinical progression.
Area of Science:
- Neuromuscular Disorders
- Genetic Diseases
- Clinical Progression Studies
Background:
- Autosomal recessive limb-girdle muscular dystrophy (LGMDR) presents with significant clinical variability.
- Understanding the progression of LGMDR, including loss of ambulation (LOA) and organ involvement, is crucial for patient management.
Conclusions:
- This study characterizes the co-occurrence of LOA, cardiac, and respiratory involvement in LGMDR.
- Findings enhance the understanding of LGMDR clinical progression across various subtypes.
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