News

January 16, 2018

AveXis Announces Expanded Clinical Development Program for AVXS-101 in Spinal Muscular Atrophy

AveXis Announces Expanded Clinical Development Program for AVXS-101 in Spinal Muscular Atrophy

AveXis, a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today provided an overview of the expanded clinical development program for the company’s initial gene therapy candidate, AVXS-101, for...

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Topics: Clinical Trials, Research, Front Page News

January 9, 2018

Scholar Rock Announces Plan to Bring Possible SMA Treatment into Clinical Trials

Scholar Rock Announces Plan to Bring Possible SMA Treatment into Clinical Trials

Scholar Rock, a biotechnology company based in Boston, has announced that they will be investing nearly $50 million to bring SRK-105, a muscle drug for possible treatment of SMA, into human clinical trials. SRK-105 is Scholar Rock’s

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Topics: Clinical Trials, Research, Front Page News

January 5, 2018

Cure SMA Launches Advocacy Action Network

Cure SMA Launches Advocacy Action Network

Working together, we’ve achieved tremendous success in advocating for ourselves, our families, and our whole community, particularly in the past few years.

Today, we’re building on this momentum with the launch of our

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Topics: Our Impact, Advocacy, Front Page News

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