Myotonic Dystrophies

Myotonic dystrophies are inherited neuromuscular disorders characterized by progressive muscular wasting (dystrophy) and muscular stiffness (myotonia). It affects both muscles and others organs such as heart, eye, breathing appartus, endocrine system, digestive tract or nervous system, these are multisystem diseases.

Clinical trials on MD ongoing at the Institute:

  • PsyDM1 : Social cognition and executive functions in pediatric DM1 disease
  • Steinert : Factors associated with hypoventilation in the myotonic dystrophy
  • PIP4 (MEX-NM-301): An Open-label, non-Comparative Study to Evaluate the Steady-State Pharmacokinetics, Safety, and Efficacy of Mexiletine in Adolescents and Children with Myotonic Disorders
  • PIP7 (MEX-NM-303): Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Mexiletine in Paediatric Patients with Myotonic Disorders Who Have Completed MEX-NM-301 Study





PIP4 (MEX-NM-301)

PIP7 (MEX-NM-303)

Intervention Mexiletine Mexiletine
Pathology DM1 DM1 Myotonic Disorders Myotonic Disorders
Principal investigator
Nathalie Angeard Thierry Perez Arnaud Isapof Arnaud Isapof
Sponsor AIM SPLF Investigator Investigator
Fundings AIM CHRU Lille
Study status Ongoing Ongoing In preparation In preparation
Recruitment status Ongoing Closed In preparation In preparation
Population Child Adult Child Child
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