Key result
Genetically confirmed dysferlinopathy exhibited high variability in disease onset, pattern of weakness, and progression rate, with no single factor accounting for this variability.
Population
193 patients with genetically confirmed dysferlinopathy
Design
Cross-sectional
Authors
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High variability in dysferlinopathy presentation warrants individualized assessment; leaves open optimal endpoints for future trials.
Cross-Sectional (n=193)
Yes
This study highlights the high clinical variability in dysferlinopathy, which is crucial for optimizing clinical care and planning future therapeutic trials.
Harris et al. (2016) conducted a cross-sectional in Dysferlinopathy (n=193). Genetically confirmed dysferlinopathy was evaluated on Baseline clinical and functional characteristics. Genetically confirmed dysferlinopathy exhibited high variability in disease onset, pattern of weakness, and progression rate, with no single factor accounting for this variability.
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