Latest News
AveXis Releases Community Statement on Expanded Clinical Trials
AveXis has provided the following community statement on AVXS-101. Dear SMA Community, At AveXis, the gene replacement therapy company developing a new approach to treat SMA known as AVXS-101, we […]
Read More ›Cure SMA to Host Webinar on Treatment Access and Clinical Trials on February 15
On Thursday, February 15, at 12:00pm CST (10:00am PST/11:00am MST/1:00pm EST), Cure SMA will hold a webinar updating the community on treatment access and clinical trials. Among the topics covered […]
Read More ›SMA Industry Collaboration Releases Spinal Muscular Atrophy Voice of the Patient (VoP) Report
Cure SMA and our partners in the SMA Industry Collaboration are pleased to announce the release of the Spinal Muscular Atrophy Voice of the Patient (VoP) Report. This report is […]
Read More ›2018 Updated SMA Drug Pipeline Released
We’ve recently released an update to the SMA drug pipeline. This latest version includes: 16 active programs, including one approved therapy. 14 pharmaceutical partners. 6 programs in clinical trials. An ever-increasing […]
Read More ›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 […]
Read More ›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-015, a muscle drug for possible treatment of SMA, into […]
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