Pipeline Pipeline Pipeline

First ever SMA regulatory filings to be submitted.

Check out our news section for the latest updates on this important milestone.

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Events Events Events

August is SMA Awareness Month.

Get involved with our annual candlelighting on the 13th, along with other planned activities, all month long!

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Conference Conference Conference

Annual SMA Conference

Check out the latest updates from the
2016 Annual SMA Conference.

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Family Support Family Support Family Support

Our community is active, engaged, and hopeful

Through information packets, care packages, local chapters, equipment, and resources, we support families today.

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Pipeline Pipeline Pipeline

We can see a future
without SMA.

We now have 18 programs in the SMA drug
pipeline. Together, we're making real
progress toward a treatment and a cure.

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2016 SMA Researcher Meeting Summary: Clinical Research Studies

2016 SMA Researcher Meeting Summary: Clinical Research Studies

We will be posting a series of summaries from our 2016 researcher meeting,...

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Cure SMA Plans a Series of Educational Modules for the SMA Community

Cure SMA Plans a Series of Educational Modules for the SMA Community

With the recent news that Biogen and Ionis will submit nusinersen for FDA...

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Cure SMA-Funded Researcher Chris Lorson  Publishes Paper in Molecular Therapy

Cure SMA-Funded Researcher Chris Lorson Publishes Paper in Molecular Therapy

Dr. Chris Lorson and his colleagues have published a paper, “

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AveXis Reports Second Quarter 2016 Interim Data from Ongoing Phase 1 Trial of AVXS-101 (Gene Therapy)

AveXis recently released...

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 Important Milestone Reached with First New Drug Program for SMA Advancing Towards Approval with the FDA

Important Milestone Reached with First New Drug Program for SMA Advancing Towards Approval with the FDA

Biogen and Ionis have been testing nusinersen in clinical trials and

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