Information on the treatment of SMA using Spinraza

Spinraza treatment for SMA at Stanford

Spinraza, the first FDA-approved drug that corrects the underlying cause of SMA, is now available commercially in the United States. The Stanford SMA program has helped investigate Spinraza’s effects, and is also helping to develop additional treatments. To find out more about our research program, or participate in studies, we encourage interested members of the SMA community to register in our research program using the contact information below.

Stanford has four years of experience using Spinraza and we are excited to now offer it clinically to patients of all ages with SMA – pending insurance approval. Individuals who want Spinraza can receive it as part of their overall care at either the pediatric or adult Stanford Neuromuscular Clinics. Stanford has weekly multi-disciplinary clinics for infants, children and adults with SMA, and now incorporates Spinraza into the comprehensive care of these patients. Both the Lucile Packard Children’s Hospital (LPCH) Pediatric Neuromuscular Clinic and the Stanford Neuroscience Health Center (SNHC) Adult Neuromuscular Clinic have accomplished teams of physicians, nurse practitioners, therapists, nurses and other providers who are experienced in the care and support of individuals affected by SMA.

The Stanford Neuromuscular Spinraza Treatment Program includes the following elements:

  • To receive Spinraza at Stanford, all individuals must be registered at, and receiving their care at, either the Stanford pediatric (LPCH) or adult (SNHC) Neuromuscular Clinic (see contact information below)
  • We assign each patient desiring Spinraza to one of 4 groups based on their exam. These are non-overlapping groups at each hospital (pediatric and adult), with each group having its own schedule for Spinraza injections:

Grp 1.      Newly diagnosed infants (at LPCH only)

-        We expect this to be the smallest group awaiting Spinraza, and anticipate being able to schedule their treatments in an ad hoc fashion that doesn’t interfere with the other groups

Grp 2.      Patients with SMA of any type in whom injections can be performed without X-ray guidance

Grp 3.      Patients with SMA of any type in whom injections require X-ray guidance

Grp 4.      Patients with SMA of any type in whom initial injections will require surgical intervention

  • We expect to provide Spinraza as part of an ongoing comprehensive treatment program in a total of 60-100 pediatric patients at LPCH, and 60-100 adult patients at SNHC
  • We expect to be able to initiate Spinraza for 8-12 new patients every 6-8 weeks at each hospital
  • We believe Spinraza will benefit all SMA patients, but cannot predict which patients might benefit most. Since we can maximally treat 12 new patients every 6 weeks (at each hospital), if more than 12 patients are awaiting treatment in any of the groups (defined above) we will randomly select next patients to start Spinraza
  • The Stanford Neuromuscular team will work with each patient and family to obtain prior authorization from the patient’s insurance, which is required before Spinraza can be administered
  • Spinraza injections follow a specific schedule

o   Patients receive 4 injections in the first 2 months, and then injections every 4 months thereafter

  • Because Spinraza takes time to work, to maximize its benefits patients must also receive clinical support meeting SMA care guidelines, as is provided at Stanford or similar multi-disciplinary neuromuscular centers.
  • All Stanford patients who receive Spinraza, whether at Stanford or another facility, are scheduled to return to our comprehensive clinic every 4 months to monitor their response to the drug and adjust their management

o   Stanford patients who start Spinraza at another institution can switch to Stanford for ongoing Spinraza injections after the patients who can only receive care at Stanford have their treatment initiated.

Contact Us (see also below)

Stanford SMA Research Program – (650) 725-4341; email address NeuromuscularResearch@stanford.edu

LPCH Pediatric Neuromuscular and SMA Clinic – New patient scheduling: (650) 723-0993, select option 3 then option 2

LPCH Pediatric Neuromuscular and SMA Clinic – Established patient scheduling: (650) 723-0993 option 3 then option 1

SNHC Adult Neuromuscular and SMA Clinic – New patient scheduling: (650) 723-6469 option 2

SNHC Adult Neuromuscular and SMA Clinic – Established patient scheduling: (650) 723-6469 option 1

Pediatric Patients

  1. If your child has previously been seen at LPCH Neuromuscular Clinic but has not had a follow-up visit in the last 6 months, please schedule an appointment by calling (650) 723-0993, select option 3, then option 1.
  2. If your child has not previously been seen at LPCH Neuromuscular Clinic, please call the new patient scheduler to make an appointment at (650) 723-0993, selecting option 3 and then option 2; have your doctor fax referrals to (650) 721-2884. Please have your doctor indicate that you are interested in Spinraza for more accurate processing.
  3. During your appointment you can: ask about Spinraza treatment for your child or alternate research options; discuss assistance options from Biogen Support Services; complete the Spinraza Start Form from Biogen.
  4. Patients already seen at the LPCH Neuromuscular Clinic in the last 6 months can fax in their Spinraza Start form (downloaded from the Biogen website) to (650) 721-6350 attention Lisa, or submit it through MyHealth, or bring it to the next appointment for finalization.
  5. Tests needed to establish eligibility for Spinraza can be performed at your clinic appointment. These tests include:  blood work, urine tests, cardiac tests, X-rays, etc.

Adult Patients

  1. If you have previously been seen at the SNHC Neuromuscular Clinic, but have not been seen in the last 6 months, please schedule an appointment by calling (650) 723-6469 and select option 1.
  2. If you have not previously been seen at the SNHC Neuromuscular Clinic, please call the New Patient Coordinators at (650) 723-6469 and select option 2; have your doctor fax in referrals to (650) 723-6002. Please have your doctor indicate that you are interested in Spinraza for more accurate processing.
  3. During your appointment you can: ask about Spinraza treatment; discuss assistance options from Biogen Support Services; and complete the Spinraza Start Form from Biogen.
  4. Tests needed to establish eligibility for Spinraza can be performed at your clinic appointment. These tests include:  blood work, urine tests, cardiac tests, X-rays, etc.

General information about drug treatment

Contact Us

If you are interested in participating in SMA research at Stanford, or want to be notified about on-going research possibilities, please register in the Stanford Neuromuscular Database by communicating with the Stanford Neuromuscular Program.

Stanford SMA and Neuromuscular Research Program – (650) 725-4341; email NeuromuscularResearch@stanford.edu

Patients with SMA who are between the ages of birth and 21 years old are welcome to receive comprehensive care in the Lucile Packard Children’s Hospital (LPCH) Pediatric Neuromuscular Clinic. If you want to be seen at the LPCH Pediatric Neuromuscular Clinic please contact us at:

LPCH (pediatric) New patient scheduling: (650) 723-0993 select option 3 then option 2
LPCH (pediatric) Established patients: (650) 723-0993 option 3 then option 1

Patients with SMA who are 21 years of age or older are welcome to receive comprehensive care in the Stanford Adult Neuroscience Health Center (SNHC) Neuromuscular Clinic. Patients aged 16-21 years of age who want to receive Spinraza treatment at SHNC, even though they are receiving their on-going care at LPCH, are also welcome to schedule an appointment in the SNHC Neuromuscular Clinic. If you want to be seen at the SNHC Adult Neuromuscular Clinic please contact us at the numbers listed below.

SNHC (adult) Established patients: (650) 723-6469 option 1 and ask to speak with the Neuromuscular Patient Care Coordinator to schedule your Spinraza evaluation appointment. As our call volume is high, you may be asked to leave a message and your call will be returned within 24 hours.

SNHC (adult) New patients: (650) 723-6469 option 2 and ask to speak with our New Patient Coordinators. You will be asked to have your current physician submit a referral and any relevant medical records such as genetic testing results. To aid in processing, your physician should state that you are interested in Spinraza treatment. Referrals should be faxed to (650) 723-6002. Once processed, our New Patient Coordinator will contact you to schedule an appointment.