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myotonic dystrophy 1

Also see myotonic dystrophy Pathology: - neurodegenerative disorder Genetics: - autosomal dominant - trinucleotide repeat (CTG) in 3' untranslated region of DM1 gene Clinical manifestations: - preferentially involves distal extremities & facial muscles - muscle weakness, facial weakness* - myotonia (impaired muscle relaxation)* - especially grip myotonia - often worse in cold weather - tapping of muscle with reflex hammer results in sustained muscle contraction [3] - no fasciculations - frontal balding - cataracts - cognitive impairment - generalized fatigue - decreased libido * myotonia may be present several years prior to muscle weakness [3] Laboratory: - DMPK genotyping - DMPK gene mutation - DMPK gene CTG repeats - DMPK phenotype - see ARUP consult [2] Special laboratory: - electrocardiogram: - arrhythmias: atrial arrhythmias, ventricular arrhythmias - heart block. first degree heart block [3] - bradycardia Complications: - diabetes mellitus type 2 - hypogonadism, testicular atrophy [3] Management: - permanent pacemaker or implantable cardioverter-defibrillator may be needed to prevent sudden death [3]

General

myotonic dystrophy; Steinert disease; myotonia dystrophica

References

  1. Medical Knowledge Self Assessment Program (MKSAP) 17, 18. American College of Physicians, Philadelphia 2015, 2018
  2. ARUP consult: Myotonic Dystrophy Type 1 (DMPK) https://arupconsult.com/ati/myotonic-dystrophy-type-1
  3. NEJM Knowledge+ Neurology
  4. Kumar A, Agarwal S, Agarwal D, Phadke SR. Myotonic dystrophy type 1 (DM1): a triplet repeat expansion disorder. Gene. 2013 Jun 15;522(2):226-30. PMID: 23570879