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Frequently Asked Questions

What should I know about the investigational medication?

The investigational medication is given in the form of an intrathecal injection, an injection into the child’s lower back that is similar to an epidural, and is designed to increase the production of SMN protein whose deficiency leads to the development of SMA disease. Researchers believe that if the children produce more of this protein, they will show improvement in symptoms of SMA.

Though the investigational medication has not been approved to treat SMA and is considered experimental, it has been studied before in infants and children with SMA and was generally well-tolerated.

However, like all medical research studies, it is possible that your child could experience side effects during this study.

What will happen during this study?

If your child is eligible to participate and you allow them to join the study, they will be randomly (like the flip of a coin) assigned to receive four injections of either the investigational medication or “sham procedure.” The “sham procedure” involves a needle prick of the skin in the area where the injection would have taken place, but the needle is not inserted deeper and no medication is injected intrathecally (into the child’s back).

There is a 66% chance your child will receive injections of the investigational medication. However, no one will know which treatment your child is receiving unless there is a medical emergency that requires this information to be provided to the study physician for appropriate medical treatment of your child.

Total study participation will last for about 16 months and include approximately 12 visits to the study clinic for treatment and evaluation of your child’s health and progress.

In addition, a study staff member will call you monthly at home throughout the study to ask questions about your child’s condition.

Do I have to participate in this study?

No, you do not have to participate. There are other options available for SMA that you can discuss with your child’s doctor.

If you do choose to participate, you may leave the study at any time and for any reason. However, if you leave the study, you will not be allowed to rejoin the study.