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Shaping the Future of Treatment

Because of the progress in our drug pipeline, we’ve launched a number of urgent initiatives to give drug programs the best chance of success.

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Events Events Events

Shaping the Future of Care

As FDA-approved treatments extend the lives of those with SMA, care will become more and more important. Our goal is not only to extend life, but also to ensure the best possible quality of life.

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The Lancet Publishes Data from Phase 2, Open-Label Trial of Spinraza (Nusinersen) in Infants

Today, Lancet published results from the Phase 2, open-label trial of nusinersen (Spinraza) in infants. This trial initiated in 2013, and...

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Annual South Florida Gala of Hope Raises over $1 Million in 6 Years

Annual South Florida Gala of Hope Raises over $1 Million in 6 Years

The 6th annual Cure SMA South Florida Chapter Gala of Hope raised a record amount of $310,000 on November 17th. Total funds raised by the...

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Annual Congressional Dinner Brings SMA Community Together on the Eve of 21st Century Cure Bill Passage

Annual Congressional Dinner Brings SMA Community Together on the Eve of 21st Century Cure Bill Passage

On Tuesday, November 29, more than 160 people from the SMA community - including government and industry partners - gathered for the 6th Annual...

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Help Us Shape the Future Together, and Reach our Goal of $750,000

Help Us Shape the Future Together, and Reach our Goal of $750,000

This has been an historic year for the SMA community. On October 28, our pharmaceutical partners at Biogen and Ionis announced that their New...

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Over 35 Cure SMA-Funded Researchers Contribute to Comprehensive SMA Encyclopedia

Over 35 Cure SMA-Funded Researchers Contribute to Comprehensive SMA Encyclopedia

Spinal Muscular Atrophy: Disease Mechanisms and Therapy, the first comprehensive encyclopedia of SMA, has been published by Elsevier....

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