Expanded Access Program (EAP) for Nusinersen in Participants With Infantile-onset (Consistent With Type 1) Spinal Muscular Atrophy (SMA)

Availability of nusinersen at an existing clinical trial site will depend on territory
eligibility. Program opening date will depend on regulatory requirements and center-specific
factors. Participating sites will be added as they apply for the EAP. A doctor must decide
whether the potential benefit outweighs the risk of receiving an investigational therapy
based on the individual patient's medical history and program eligibility criteria

Key Inclusion Criteria:

- Genetic documentation of 5q SMA homozygous gene deletion, homozygous mutation, or
compound heterozygote.

- Onset of clinical signs and symptoms at ≤ 6 months (180 days) of age, consistent with
infantile onset, Type I SMA

- Patient whose care in the opinion of the treating physician meets, and is expected to
continue to meet, the guidelines set out in the 2007 Consensus Statement for Standard
of Care in SMA

Key Exclusion Criteria:

- Patient is qualified to participate in an ongoing clinical trial with nusinersen

- Participation in a prior nusinersen study

- Previous exposure to nusinersen

- History of brain or spinal cord disease that would interfere with the LP procedures
or CSF circulation

- Presence of implanted shunt for the drainage of CSF or implanted CNS catheter

- Previous or current participation in a clinical trial with an investigational gene
therapy for SMA

- Participation in a study with an investigational therapy for SMA within 6 months or
five half-lives of the investigational drug, whichever is the longer, prior to the
first dose of nusinersen.

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply