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. Stanford has over four years of experience using Spinraza and we are excited to offer it to people of all ages. Stanford was the first to treat an infant in 2013, and since FDA approval of Sprinraza in December 2016, we completed 3 of the first 6 commercial treatments of children and we treated the first adult. The Stanford SMA program was at the forefront of clinical trials for Spinraza, and is involved with the development of additional motor neuron treatments. To learn more about our research program, or to participate in studies, please register on our research program mailing list by emailing NeuromuscularResearch@stanford.edu and requesting a database consent form.
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, social workers 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 prior authorization from the patient’s insurance, which is required before Spinraza can be administered
- Spinraza injections follow a specific schedule. Patients receive 4 injections in the first 2 months as loading therapy. Maintenance therapy consists of one injection every 4 months.
- 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
LPCH Pediatric Neuromuscular and SMA Clinic – New patients call: (650) 723-0993, select option 3 then option 2
LPCH Pediatric Neuromuscular and SMA Clinic – Return patients call: (650) 723-0993 option 3 then option 1
SNHC Adult Neuromuscular and SMA Clinic – New patients call: (650) 723-6469 option 2. 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 can be faxed to (650) 723-6002. Once processed, our New Patient Coordinator will contact you to schedule an appointment.
Those 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 number below.
SNHC Adult Neuromuscular and SMA Clinic – Return patient scheduling: (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.
Stanford SMA Research Program - 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. (650) 725-4341; email address NeuromuscularResearch@stanford.edu
Pediatric Patient Appointments
- 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.
- If your child has not been seen at the 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.
- During your appointment, 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 (given to you in clinic).
- 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 Patient Appointments
- 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.
- If you have not previously been seen at the SNHC Neuromuscular Clinic, have your doctor fax a referral to (650) 723-6002. Please have your doctor indicate that you are interested in Spinraza for more accurate processing. You may call the New Patient Coordinators at (650) 723-6469 option 2 to schedule.
- During your appointment you can expect to receive more information about Spinraza. Your visit may include a consultation with one of our neuromuscular MDs or NP, baseline physical therapy assessment, baseline breathing test, labs, and imaging of your spine. Treatment is dependent on our ability to access to your spinal canal through your lower back, which can be impacted by spinal fusion.