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

Register for a fall event today!

Check out our events calendar to see a full
list of events happening in your area!

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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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Roche Launches New Phase 2 Clinical Trials

Roche has officially launched two Phase 2 clinical trials testing RO7034067 (also known as RG7916). The trials will begin recruitment in...

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Catch Up on Research News from the 2016 Family Friendly Research Poster Session

Catch Up on Research News from the 2016 Family Friendly Research Poster Session

Over the last decade, we’ve seen many advances in spinal muscular atrophy research, from new techniques in gene therapy to drugs that show...

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Roche Provides New Update on Clinical Development of RG7916

Roche, PTC Therapeutics, and the SMA Foundation have released a community update on the development of their SMN2 splicing modifier clinical...

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2016 SMA Researcher Meeting Summary: Regulation of SMN Protein Expression and Function

2016 SMA Researcher Meeting Summary: Regulation of SMN Protein Expression and Function

The SMA Researcher Meeting is the largest research meeting in the world...

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Cure SMA Welcomes Aboard New Board and Committee Members

Recently, Cure SMA confirmed three new board members and one new committee member. These new members will provide additional leadership in the...

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