Global Open-Label Extension Study of Del-desiran for the Treatment of DM1

This study is mainly for people who have already been part of a previous study with the medicine AOC 1001 (now called del-desiran). To join, you would need to have completed your previous study well, followed all the instructions, and your doctor thinks this study is right for you.

There's also a small group for people who haven't taken del-desiran before. For this group, you'd need a confirmed diagnosis of Myotonic Dystrophy Type 1 (DM1) and 'myotonia' in your hands, which means your muscles might struggle to relax after tensing.

In general, you wouldn't be able to join if you are pregnant, breastfeeding, or planning to get pregnant. You'd also need to be willing to use contraception if necessary. For the new group, there are extra things that might stop you from joining, such as certain uncontrolled health conditions like diabetes, recent heart problems, or if you're taking other experimental medicines. Your doctor will discuss all these details with you.

If you join this study, you'll be taking part for about four years and two and a half months (about 50 months). You'll receive the study medicine, del-desiran, through a drip into your arm (an 'intravenous infusion') once every eight weeks. This means you'll have seven doses each year. The last dose will be roughly four years into the study, and then you'll have one final check-up about eight weeks after that last dose.

During your visits, the research team will regularly check your health, making sure the medicine is safe for you and seeing how well it's working. This will likely involve blood tests, physical exams, and other assessments related to your DM1.