Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials

This is a global, prospective, multi-center study that is designed to assess the
long-term safety and efficacy of OAV101 in patients who participated in an OAV101
clinical trial. The assessments of safety and efficacy in Study COAV101A12308 will
continue for 15 years from the date of OAV101 administration in the previous clinical
trial. The study is comprised of a Baseline Period and 3 Follow-up Periods. Follow-up Periods 1
and 2 consist of in-person visits and Period 3 consists of tele-visits. Follow-up Periods
1 and 2, which includes Baseline through Year 5 visits, assessments will be performed at
the Investigational site. During Follow-up Period 3 (Year 6 to up to Year 15 after OAV101
administration), participants/caregivers will be contacted using tele-visits annually for
remote assessments. All patients will enter the study at the baseline visit and continue
until 15 years since OAV101 administration is reached. Total duration of participation in
the study will be dependent upon time of enrollment relative to OAV101 administration and
will vary by participant.