Latest News
AveXis Announces the Completion of Enrollment for Phase 1 Gene Therapy Trial
AveXis has announced that they have completed enrollment in a Phase 1 study testing systemic delivery of gene therapy for SMA. The trial has enrolled a total of 15 patients […]
Read More ›Cytokinetics Opens Enrollment for Phase 2 Trial
Cytokinetics has opened enrollment for a Phase 2 study testing CK-2127107 in teens and adults with SMA type II, III or IV. We are excited to see the continued progress […]
Read More ›Cure SMA Funding Leads to 18 New Research Publications in 2015
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 receive Cure SMA funding often […]
Read More ›Cure SMA Funds 28 Research Projects in 2015, as Drug Pipeline Grows to 18 Programs
Our approach to research is focused on continually expanding the pipeline of potential drugs and therapies for SMA, cultivating new approaches and advancing promising ideas. Every year, the pipeline gets […]
Read More ›Cure SMA Reaches Thousands of Families in 2015
Each year, Cure SMA provides thousands of families with vital support and resources to help them live active, engaged and hopeful lives. Our programs provide tangible support for families affected […]
Read More ›5th Annual Congressional Dinner Brings Together Families, Government and Industry
On December 1, more than 120 people from the SMA community—including government, and industry partners—gathered for the 5th Annual “Hope on the Hill” Congressional Dinner. Several Members of Congress attended […]
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