News

December 23, 2016

FDA Approves Spinraza for SMA

FDA Approves Spinraza for SMA

On December 23, the FDA announced that it has approved SpinrazaTM (nusinersen) to treat spinal muscular atrophy, making it the first-ever FDA-approved therapy for SMA.

We...

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Topics: Our Impact, Clinical Trials, Advocacy, Front Page News

December 21, 2016

AveXis to Use Intended Commercial GMP Product in SMA Type 2 Study

AveXis, Inc., a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-­threatening neurological genetic diseases, today reported that, based on emerging data from its intended...

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Topics: Clinical Trials, Front Page News

December 19, 2016

Cure SMA Participates in a Special FDA Listening Session

Cure SMA Participates in a Special FDA Listening Session

Last week, Cure SMA participated in a listening session with FDA Commissioner Robert M. Califf, MD, MACC. The FDA organized the listening session in order to gain a better understanding of the current goals, priorities, and challenges of our community.

Representatives from Cure SMA...

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Topics: Community & Awareness, Our Impact, Advocacy, Front Page News

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