Research
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 […]
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 […]
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 […]
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 […]
Read More ›Cure SMA Announces Funding for Care Centers Across the US
Cure SMA is pleased to announce the first grants from $450,000 in funding to help increase capacity at SMA treatment sites across the US. In […]
Read More ›Biogen and Ionis Enter Into New Collaboration to Identify Novel Therapies for the Treatment of Spinal Muscular Atrophy
Biogen and Ionis Pharmaceuticals announced that they have entered into a new collaboration agreement to identify new antisense oligonucleotide drug candidates for the treatment of […]
Read More ›2017 Research Year-in-Review
Over our past fiscal year—from July 1, 2016, to June 30, 2017—Cure SMA has funded 28 projects with over $2.5 million in new research funding. […]
Read More ›AveXis Announces Plan to Initiate Phase 1 Trial in SMA Type 2 Utilizing Intrathecal Delivery of AVXS-101
AveXis, Inc., a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced the U.S. Food and […]
Read More ›Cure SMA-Funded Research Results in 21 Published Journal Articles
An important goal of our research funding strategy is to share scientific findings with the broader scientific community. To help accomplish this goal, scientists who […]
Read More ›Cytokinetics Publishes Clinical Trial Data for CK-2127107
Cytokinetics, Inc. recently announced the publication of results from three early clinical trials in healthy volunteers that evaluated safety, tolerability, pharmacokinetics, and pharmacodynamics of CK-2127107. […]
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