Latest News
AveXis Announces Single-Arm Design for Pivotal Study 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 that the planned pivotal study of AVXS-101 in spinal […]
Read More ›Family Registration for the 2017 Annual SMA Conference is Now Open
Registration is now open for the 2017 Annual SMA Conference! The conference will be held at Disney’s Contemporary Resort at Disney World in Orlando, FL, from Thursday, June 29, through […]
Read More ›Spotlight Innovation Launches Development of STL-182 to Treat SMA
Spotlight Innovation has obtained an exclusive, worldwide license to STL-182, a novel compound invented by Drs. Elliot Androphy at Indiana University and Kevin Hodgetts, director of the Laboratory for Drug […]
Read More ›6th Annual Congressional Dinner Furthers SMA Advocacy Work
This year’s 6th Annual “Hope on the Hill” Congressional Dinner will be held on Tuesday, November 29, bringing together families, government, and industry for an important evening of collaboration. We […]
Read More ›Cure SMA-funded Researcher Sara Custer Publishes Paper
Sara Custer, PhD, and her colleagues at Indiana University have published a new paper, “Altered mRNA Splicing in SMN-Depleted Motor Neuron-Like Cells” in the journal PLOS ONE . The research […]
Read More ›SMA Community is Granted a Patient Focused Drug Development Meeting with the FDA
Cure SMA is excited to announce that the SMA community has been granted a Patient Focused Drug Development Meeting with the FDA. At the Patient Focused Drug Development (PFDD) Meeting, […]
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