FDA Approves Medication to Treat Spinal Muscular Atrophy

On December 23, the FDA announced that it has approved Spinraza^TM (nusinersen) to treat spinal muscular atrophy, making it the first-ever FDA-approved therapy for SMA.

We are thrilled to see our community’s efforts culminate in the approval of Spinraza: not only the first-ever approved treatment for this disease, but also one that addresses the underlying genetic cause of SMA. This has been a story of all groups—families, researchers, companies and the FDA—working together as one community to reach this amazing milestone.

The approval from the FDA for all SMA—pediatric and adult—is the broadest possible label, with no restrictions—and this matches our core value at Cure SMA of being one united community for all ages and all types of SMA.

Written by Joan Vaughn

I’m Joan, and I know home care from the inside out. I’ve navigated my daughter through 24/7 home care nursing and managed her PCA’s. I’ve helped my parents move into (and out of) assisted living, Medicare episodes, skilled nurse visits, home health aids, and PCAs. Sandwiched in all of that, I’ve helped coordinate home care equipment and supplies, physical therapists, occupational therapists, county workers, and medications (oh, the medications). I know emergency departments, children’s hospitals, cancer wards, cardiac floors, and specialty units. I can recite health insurance policies (and call their bluff). I know a lot of other things in life too, like Shakespeare, where to sit to see a great sunset over a lake, how to keep myself healthy, how to read a profit/loss statement and state statutes, when to use a comma, and that gratitude is one of the keys to success. So I’m going to have this blog and write about home care from time to time. I’ll include topics like current legislation, professional boundaries in a tricky environment, and being a caregiver. I’ll also post some guest blogs from clients and nurses. Join me in the conversation.

View all posts by: Joan Vaughn

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