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.
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.
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.
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.
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.
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 800-572-1717. 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.