Information on the treatment of SMA using Spinraza

Spinraza Treatment for SMA at Stanford

Spinraza, is the first FDA-approved drug that increases SMN protein content in motor nerve cells; deficiency of SMN in these cells is the underlying cause of SMA. Spinraza is now available commercially throughout the United States. The Stanford SMA program continues to investigate Spinraza in pediatric trials and is currently studying its effects in adults with SMA. Additionally, we are investigating other ways to increase SMN production in neurons, and we are working to determine the benefits of combining SMN increases with other therapies that can optimize strength and function.. To find out more about our research program, or to participate in studies, we encourage interested members of the SMA community to register in our research program at: https://med.stanford.edu/day-lab/recruitment.html.

Stanford has six years of experience using Spinraza and we are excited to offer it clinically to patients of all ages with SMA – pending insurance approval. Individuals who want Spinraza may 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 Pediatric Neuromuscular Clinic at Lucile Packard Children’s Hospital (LPCH) and the Adult Neuromuscular Clinic at the Stanford Neuroscience Health Center (SNHC) have accomplished teams of physicians, nurse practitioners, therapists, nurses, pharmacists 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)
  • The Stanford Neuromuscular team will work with each patient and family to obtain required prior authorization from the patient’s insurance.
  • Spinraza injections follow a pre-specified schedule
    • Patients receive 4 injections in the first 2 months, and then injections every 4 months thereafter
  • Because Spinraza takes time to work, to maximize the possible benefits of Spinraza, all patients must receive clinical care that  meets SMA care guidelines 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. During these clinic visits, patients will be evaluated using standard tests to measure the response to Spinraza. These assessments are required by insurance companies in all patients receiving Spinraza; lack of these assessments will result insurance companies cancelling authorization for continued access to Spinraza.

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. To assure rapid procession, please tell the scheduler, and have your doctor indicate, that your child has SMA and you are interested in Spinraza.
  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 Jessica, 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, review of genetic results, 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, review of genetic results, 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 18 years of age or older are welcome to receive comprehensive care in the Stanford Adult Neuroscience Health Center (SNHC) Neuromuscular Clinic. If for particular reasons patients aged 16-21 years of age want to receive Spinraza treatment at SHNC, even though they are receiving their on-going care at LPCH, they are welcome to schedule an appointment in the SNHC Neuromuscular Clinic or to talk with the LPCH Neuromuscular Team regarding their preferences. 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.