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Myotonic Dystrophy

Neuromuscular disorder characterized by PROGRESSIVE MUSCULAR ATROPHY; MYOTONIA, and various multisystem atrophies. Mild INTELLECTUAL DISABILITY may also occur. Abnormal TRINUCLEOTIDE REPEAT EXPANSION in the 3' UNTRANSLATED REGIONS of DMPK PROTEIN gene is associated with Myotonic Dystrophy 1. DNA REPEAT EXPANSION of zinc finger protein-9 gene intron is associated with Myotonic Dystrophy 2.

Drugs by status

Phase 3 trials (5)

  • dehydroepiandrosterone
  • methylphenidate
  • mexiletine
  • tideglusib
  • aoc 1001

Phase 2 trials (8)

  • pitolisant
  • mecasermin
  • ionis-dmpkrx
  • ranolazine
  • insulin-like growth factor-binding protein-3
  • atx-01
  • pgn-edodm1
  • sar446268

Phase 1 trials (3)

  • amlodipine
  • flumazenil
  • aro-dm1

Other trials (2)

  • caffeine
  • theobroma cacao whole

Organizations

Organization Involved with Phase 3 Indications (6)

  • Lupin
  • AMO Pharma Limited
  • University of Versailles
  • Laval University
  • University of Copenhagen
  • University of Paris

Organization Involved with Phase 2 Indications (8)

  • Gilead Sciences
  • Ionis Pharmaceuticals, Inc.
  • Muscular Dystrophy Association
  • National Institute of Neurological Disorders and Stroke (NINDS)
  • Newcastle University
  • Ohio State University
  • University of Rochester
  • Insmed, Inc.

Organization Involved with Phase 1 Indications (2)

  • University of California, San Francisco
  • Expansion Therapeutics, Inc.

ICD-10 crosswalk

  • G71.11 — Myotonic muscular dystrophy

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