ELX1019 Is an Investigational Therapeutic for People Living with Myotonic Dystrophy Type 1 (DM1)
What is Myotonic Dystrophy Type 1 (DM1)?
DM1 is a rare, inherited muscle-wasting disease that affects tens of thousands of people around the world. It causes progressive muscle weakness, fatigue, breathing and heart complications, and cognitive difficulties — often worsening over time. There is currently no cure.
Estimated to affect over 40,000 people in the U.S. and more than 74,000 in Europe.
When Does It Start?
Symptoms can appear at any age, from birth to adulthood. Most people with adult-onset DM1 begin experiencing symptoms between their teens and 50s.
What Causes DM1?
DM1 is caused by a genetic defect in the DMPK gene, which contains an abnormal expansion of DNA known as a CTG repeat. This expanded sequence leads to the production of toxic RNA in cells. The toxic RNA interferes with normal gene function by disrupting RNA splicing — a process essential for creating healthy proteins — which in turn leads to many of the wide-ranging symptoms seen in DM1.
Common Symptoms
Across the Body
Severity and symptoms vary, even among family members.
Is There a Treatment?
There is no disease-modifying treatment for DM1 yet.
Proof-of-Concept Model
The overlap between DM1 pathology and aging biology positions DM1 as an ideal testbed for evaluating therapies targeting age-related mechanisms. Treatments that restore RNA processing, reduce senescent cell burden, or enhance mitochondrial function may hold promise across age-associated conditions.