Population
Patients with dysferlinopathies (Miyoshi myopathy and limb girdle muscular dystrophy type 2B)
Design
Review
Authors
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Should not yet change practice in dysferlinopathies; leaves open whether exon-skipping or minigene transfer will prove effective in trials.
Exon-skipping and minigene transfer strategies represent promising preclinical therapeutic approaches for dysferlinopathies.
Barthélémy et al. (2011) studied this question.
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