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We can see a future without SMA.

SMA is the number one genetic cause of death for infants. But together, we're making real progress toward a treatment and cure.

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Make History With Your Gift This Year-End

Our goal is to raise $540,000 before December 31 to fund even more breakthroughs next year and beyond.

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SMA Drug Pipeline Continues to Grow

We now have 17 active drug programs in the pipeline, including six in clinical trials.

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Together, We Can Cure SMA

Cure SMA is our name, and it’s also our rallying cry. Show our collective strength, united to cure SMA.

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Cure SMA Updates

Isis Expands Phase 3 Clinical Trials

Isis recently announced that they have tested the first patient in CHERISH, a multi-center Phase 3 clinical study...

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Isis recently announced that they have tested the first patient in CHERISH, a multi-center Phase 3 clinical study...

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Cure SMA Researchers Present at Society for Neuroscience Meeting

Cure SMA Researchers Present at Society for Neuroscience Meeting

PTC Therapeutics Announces New Phase 1a/2b Clinical Trial

PTC Therapeutics, Inc. announced today that its joint development program in Spinal Muscular Atrophy (SMA) with Roche and the SMA Foundation (SMAF) has...

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PTC Therapeutics, Inc. announced today that its joint development program in Spinal Muscular Atrophy (SMA) with Roche and the SMA Foundation (SMAF) has...

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