News

September 29, 2017

AveXis Announces Plan to Initiate Pivotal Trial of AVXS-101 in SMA Type 1 Using Product from New GMP Commercial Process

AveXis Announces Plan to Initiate Pivotal Trial of AVXS-101 in SMA Type 1 Using Product from New GMP Commercial Process

AveXis, Inc., a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced...

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Topics: Clinical Trials, Front Page News

September 28, 2017

Cure SMA Contributes Article to a Special Issue of the Journal Gene Therapy

To celebrate the FDA approval of Spinraza, Cure SMA contributed an article to the September special issue of the journal Gene Therapy. The issue , “Spinraza and Advanced Therapies: a stakeholder special,” covers a variety of perspectives and includes comments and review papers from SMA research funders, people affected by SMA,...

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Topics: Our Impact, Research, Front Page News

September 20, 2017

Newborn Screening Advocacy Update

Newborn Screening Advocacy Update

At our Annual SMA Conference in July, we announced a grassroots campaign to implement newborn screening for SMA in all 50 states. We know that newborn screening and early treatment is the best chance we have to change the course of SMA for the next generation and beyond. We challenged our...

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Topics: Community & Awareness, Advocacy, Front Page News

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