What is the purpose of the STEER Study?
The purpose of the STEER Study is to understand if the investigational gene therapy OAV101 is safe and can help improve muscle function in children aged 2 to less than 18 years with Type 2 SMA.
What is OAV101?
OAV101 is a gene therapy designed to deliver a new functioning copy of the SMN1 gene to the motor neurons. The goal is for the new SMN1 gene to make enough SMN protein to prevent further loss of motor neurons.
Who can participate in this study?
The study is for children aged 2 to less than 18 years old with Type 2 SMA who:
- Are able to sit independently but have never been able to walk independently.
- Began showing symptoms of SMA at 6 months of age or older.
- Have not yet received any treatments that increase SMN.