Eine Studie zur Untersuchung der Sicherheit und Wirksamkeit von RO7204239 in Kombination mit Risdiplam (RO7034067) bei Teilnehmern mit Spinaler Muskelatrophie

To join this study, people must have a confirmed genetic diagnosis of Spinal Muscular Atrophy (SMA) and show symptoms of the condition. The study is open to both boys and girls.

For the first part of the study, depending on the group, participants can be between 2 and 10 years old. For the second part, participants can be between 2 and 25 years old and must be able to walk unassisted (without crutches, walkers, or any help) for about 10 meters in 30 seconds or less. A specific group in the first part will include young people aged 5-10 who are not able to walk but can sit up on their own for a short time.

People who have received other SMA treatments before might be able to join, but there are specific waiting periods mentioned for each previous treatment. For example, if you've had gene therapy (like onasemnogene abeparvovec), you would need to wait at least 90 days. If you are currently taking risdiplam, you might be able to switch to the study's version of the medicine.

Taking part in this study would involve regular visits to the clinic for assessments and to receive the study medicines. You would either receive RO7204239 combined with risdiplam or a placebo (a dummy medicine) combined with risdiplam, depending on the study group you are in. Doctors and nurses will monitor your health closely, perform examinations, and take blood samples. They will also assess your muscle strength and movement abilities using specific tests. The exact number of visits and the total length of your participation would be discussed in detail by the study team.