About the Investigational Drug

Nusinersen is designed to increase the production of SMN, a protein that is needed for nerve and muscle function.


Nusinersen is what is known as an “antisense oligonucleotide.”
Talk to your doctor to learn more.

The Investigational
Drug Nusinersen is:

  • A type of therapy that targets a specific gene and is believed to increase its protein production
  • Delivered by injection into the spinal canal every four months (following four initial loading doses over a two-month period)
  • This clinical research study aims to study how children respond when they receive nusinersen following onasemnogene abeparvovec treatment.

Administration of the Investigational Drug

All study participants will receive nusinersen. It is administered through an injection, using a thin needle, into a fluid-filled space (called the spinal canal) at the base of the spinal cord (in the lower back). This type of procedure is called a lumbar puncture. During the lumbar puncture, the study doctor will also remove a small amount of cerebrospinal fluid (CSF) for testing.

Please watch the video for general information about lumbar punctures and CSF collection. For more detailed information on the study injection, please speak with a member of the study team.