About the Study Medication

What is DM1?

Our genes provide instructions (genetic code) to our cells to make proteins that play important roles in our bodies. People with myotonic dystrophy type 1 (DM1, also known as Steinert Disease) have a mutation (or error) in their genetic code in a gene called myotonic dystrophy protein kinase (DMPK). This mutation prevents the body from correctly processing certain proteins that are important for muscle, respiratory, cardiac health, and brain function. Many types of proteins can be affected, accounting for the wide variety of possible DM1 symptoms.

What is PGN-EDODM1?

PGN-EDODM1 is an investigational drug designed to address the root cause of DM1 and restore the body’s ability to correctly process the proteins affected by DM1. This restoration may slow, halt or reverse DM1 symptoms.

Will I receive the study medication?

In this study, either PGN-EDODM1 or placebo will be given as a single IV infusion (through a needle in a vein), which takes about an hour. You will have a 3 out of 4 chances of receiving PGN-EDODM1 instead of placebo. Neither you nor the study staff will know which you have received.

What is an “investigational medicine?”

An investigational drug is a substance that is being tested in clinical research studies and may or may not be approved by the regulatory authorities (e.g. U.S. Food and Drug Administration, Medicines and Healthcare products Regulatory Agency or Health Canada) for treatment of this condition. Researchers compare the investigational drug to a placebo. A placebo is a substance that has no active drug in it.