Study of intravenous AOC 1001 to evaluate long-term safety and effectiveness in patients with myotonic dystrophy type 1

This study focuses on Myotonic Dystrophy Type 1, also known as DM1, which is an inherited muscle disorder that causes progressive muscle weakness and affects multiple body systems. The study will test a medication called AOC 1001, which is given through an intravenous infusion (delivered directly into a vein). This medication is a specialized type of drug that combines an antibody with genetic material designed to target the underlying cause of the disease.

The purpose of this research is to evaluate how safe and well-tolerated AOC 1001 is when used as a long-term treatment for people with Myotonic Dystrophy Type 1. During the study, participants will receive regular infusions of the medication over an extended period. The medication will be administered at a dose based on the participant's body weight, up to a maximum of 4 mg per kilogram.

This is a continuation study, which means it follows up on previous research with the same medication. The study is "open-label," which means all participants will receive the active medication, and there is no placebo group. The treatment period may last up to 48 weeks, during which participants will receive multiple doses of the study medication while being monitored for any effects or side effects.