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Spinraza is Approved!

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New Hope for Kids and Adults with Spinal Muscular Atrophy

For more than 65 years, MDA has been on the frontlines of research for spinal muscular atrophy (SMA) and other neuromuscular diseases. We funded foundational work in SMA and invested in the early-stage development of nusinersen (brand name Spinraza). Today we celebrate FDA approval of this promising drug.

This advancement could not have been made possible without the amazing support of the MDA community over the years. Thank you to all who walked, ran, fundraised, dropped a dollar in a fireman’s boot, texted in a telethon donation or raised awareness online. You have made this life-changing achievement possible!

 

  • 5 years ago

    There were no approved therapies to treat the underlying cause of SMA

  • September 2016

    In partnership with Ionis Pharmaceuticals, Biogen submitted a New Drug Application for Spinraza

  • December 2016

    Spinraza is granted FDA approval to treat kids and adults with SMA

Your story matters

We hope you are as excited about this news as we are. If you'd like to share your story with Spinraza or thoughts on the impact this will make, we'd love to hear it.

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Articles & Stories

  • FDA Approves First-Ever Treatment of SMA

    On Dec. 23, 2016, the SMA community achieved a goal that would not have been possible without the support and dedication of the families, physicians and researchers who have passionately pursued a treatment for spinal muscular atrophy (SMA). We are proud to announce that SPINRAZA™ (nusinersen) has been approved by the FDA.

    Read More

  • Encouraging Results in Nusinersen (Spinraza) Trial for Type 2 SMA

    Spinraza is a disease-modifying antisense drug, one of a class of experimental therapeutic molecules designed to target genetic instructions at the RNA stage (an intermediate step between DNA and protein manufacturing stage inside cells). It is designed to increase production of the needed SMN protein.

    Read More

  • SMA Research: The Curious Case of a Backup Gene

    For Adrian Krainer at Cold Spring Harbor Laboratory on New York’s Long Island, the preferred strategy to find a cure for SMA involves changing the SMN2 gene’s instructions by altering a process called splicing, a kind of molecular cutting and pasting that cells perform.

    Read More

And this is just the beginning

Seeing this treatment become an available option for our families is a significant step forward - one that we know will spark more innovation and drug development across diseases. There are many other promising therapies moving closer to the finish line, but we can't keep this momentum going without you.

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MDA Resource Center: We’re Here For You

Our trained specialists are here to provide one-on-one support for every part of your journey. Send a message below or call us at 1-833-ASK-MDA1 (1-833-275-6321). If you live outside the U.S., we may be able to connect you to muscular dystrophy groups in your area, but MDA services are only available in the U.S.

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