Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.
Questions? Read on to learn more about SPINRAZA and the support services available to you. Please remember that your doctor should be your primary resource for any medical questions. Need additional support?
Call 1-844-4SPINRAZA (1-844-477-4672), Monday through Friday, from 8:30 AM—8:00 PM ET.
Spinal muscular atrophy (SMA) is a genetic disorder that is caused by a lack of survival motor neuron (SMN) protein, a protein that’s key for muscle development and movement.
SMA starts in the central nervous system (CNS) and affects all the muscles in the body. It’s what’s known as a neuromuscular disease because it affects the motor neurons that power muscles. Due to the degenerative nature of the disease, people with SMA will experience a decline in muscle strength over time, although the rate and severity can vary among individuals.
SPINRAZA is the first approved therapy for pediatric and adult patients with SMA.
The effectiveness of SPINRAZA was established in the longest clinical trial program in SMA to date, which included the pivotal ENDEAR and CHERISH clinical trials. The ENDEAR pivotal study studied survival without permanent ventilation in 121 children with early-onset SMA. The CHERISH clinical trial studied changes in motor function in 126 nonambulatory individuals with later-onset SMA.
In clinical studies, the most common side effects of SPINRAZA included lower respiratory infection, fever, constipation, headache, vomiting, back pain, and post-lumbar puncture syndrome (headache related to the intrathecal procedure).
Because an increased risk of bleeding and kidney damage has been seen in similar medications, individuals taking SPINRAZA may be at similar risk.
It is recommended that your healthcare provider (HCP) perform blood and urine testing once before treatment and again before each dose to monitor for signs of these risks.
SMA is a disease of the central nervous system (CNS), caused by an insufficient amount of SMN protein in motor neurons. SPINRAZA is delivered directly to the CNS, where it increases SMN production, targeting an underlying cause of SMA.
SPINRAZA is given as an injection into cerebrospinal fluid (the fluid in the spine) during a medical procedure called an intrathecal injection, an established route of medication delivery used to target the CNS.
We are committed to making sure that individuals treated with SPINRAZA and their families are able to access treatment.
We have a user-friendly search engine on our website that can help you find the HCPs and treatment facilities closest to you that may be able to administer SPINRAZA. The search results show an interactive map with information about HCPs and facilities that have chosen to be listed.
Before choosing a treatment center, be sure to speak with your primary HCP.
Yes. Once you or your child is prescribed SPINRAZA, you can learn about and access SMA360°™* support services from Biogen. Biogen’s SMA360° support program provides certain services that address nonmedical barriers to access, including financial concerns and insurance coverage.
*SMA360° patient services from Biogen are available only to those who have been prescribed SPINRAZA.
SMA360° is intended for US residents only.