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: Identification of Candidate Therapeutic Targets and Modifiers, Part 1

2016 SMA Researcher Meeting Summary: Identification of Candidate Therapeutic Targets and Modifiers, Part 1

The SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA. This year we had a record setting 350...

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Cure SMA Recognizes Judith Melki on the Anniversary of the Discovery of the SMN Gene

Cure SMA Recognizes Judith Melki on the Anniversary of the Discovery of the SMN Gene

During the 2016 SMA Researcher Meeting, the anniversary of the identification of...

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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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