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Title
Myotonic Dystrophy Type 1
ImagePath
System
trinucleotide repeat expansion
Overview
AD CTG repeat expansion in DMPK gene causing myotonia & multisystem involvement.
Pathophysiology
CTG expansion → abnormal RNA binding proteins → splicing defects (eg, CLCN1) → myotonia, endocrine issues.
Presentation
Difficulty releasing handshake (grip myotonia), cataracts, balding, testicular atrophy, arrhythmias.
Diagnosis
PCR repeat sizing; electromyography: myotonic discharges.
Management
Mexiletine for myotonia, slit‑lamp for cataracts, pacemaker for heart block.
Epidemiology
1 : 8000; anticipation via maternal transmission.
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