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:

• DYNE101-DM1-201: A Randomized, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-101 Administered to Participants with Myotonic Dystrophy Type 1
• 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
• ArthemiR CT-ATX-01-DM1-1.1: A Phase 1/2a Double-Blind, Placebo-controlled, Single- and Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Clinical Efficacy of Intravenous Administration of ATX-01 In Male and Female Participants aged 18 to 64 with Classic Myotonic Dystrophy Type 1 (DM1)
• Vertex VX23-670-001: Phase 1/2, Randomized, Double-blind, Placebo‑controlled Single- and Multiple‑dose, Escalation Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics ofVX‑670 in Adult Subjects with Myotonic Dystrophy Type 1
• Avidity AOC 1001-CS3: A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1

Contact : essais-adultes@institut-myologie.org