Spinraza (nusinersen), marketed by Biogen, was the first FDA-approved therapy to treat SMA. It is an SMN-enhancing therapy that works by targeting the SMN2 gene, causing it to make more complete protein.

More About Spinraza

  • Spinraza is FDA-approved for all ages and types of SMA.

  • Spinraza is given via an intrathecal (IT) injection, which is an injection into directly into the cerebrospinal fluid through the lower back. Individuals receive four “loading doses” within the first 2 months of treatment. Once those loading doses are completed, they receive a maintenance dose every 4 months for the duration of the individual’s life.

  • Spinraza is an antisense oligonucleotide. Antisense drugs are small snippets of synthetic genetic material that bind to ribonucleic acid (RNA), so they can be used to fix splicing errors in genes such as SMN2.

  • As with all treatments, timing is critical. Individuals who began Spinraza as soon after diagnosis as possible had better results than those who waited to begin treatment.

  • Several sites across the U.S. are dosing individuals with Spinraza. A number of these sites can be found on our map locator tool.

  • To find insurance policies and payer information for Spinraza, please click here.

  • Check out Biogen’s patient brochures on Spinraza for pediatrics and adults.

History of Spinraza

From 2003 to 2006, Cure SMA provided the very first research funding needed to begin investigation into this therapeutic approach. We would like to thank and acknowledge Cold Spring Harbor Laboratory (CSHL) and the University of Massachusetts Medical School for generating critical intellectual property for the program that was licensed to Ionis Pharmaceuticals.

We specifically thank Drs. Adrian Krainer, Yimin Hua, and colleagues at CSHL for years of dedication to and hard work on the pre-clinical development of Spinraza for SMA, as well as Drs. Ravindra Singh and Elliot Androphy for their work, funded by Cure SMA, in originally identifying the ISSN1 gene sequence, which is the sequence targeted in Spinraza.

After the pre-clinical work and early clinical testing was complete, Ionis Pharmaceuticals partnered with Biogen to launch a number of clinical trials testing Spinraza.

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