Front Page News
Cure SMA Joins SMA Europe and TREAT-NMD for Meeting with EMA Regulators
Cure SMA will be participating in a one-day workshop with The European Medicines Agency (EMA—the European equivalent of the FDA), SMA Europe and TREAT-NMD to […]
Read More ›Cure SMA to Host Webinar on the New Drug Application Process
On Tuesday, December 6, Cure SMA will be hosting a one-hour webinar on the New Drug Application (NDA) process. The webinar will be held at […]
Read More ›Biogen and Ionis Community Statement on Interim Analysis of CHERISH (Type 2) Trial
Biogen and Ionis Pharmaceuticals have provided the following community statement regarding the interim analysis of the CHERISH trial: Dear Members of the SMA Community, Today […]
Read More ›Biogen and Ionis Pharmaceuticals Announce SPINRAZA (nusinersen) Meets Primary Endpoint at Interim Analysis of Phase 3 CHERISH Study
Biogen and Ionis Pharmaceuticals announced that SPINRAZA (nusinersen), an investigational treatment for spinal muscular atrophy (SMA), met the primary endpoint at the interim analysis of […]
Read More ›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 […]
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, […]
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 […]
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 […]
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 […]
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 […]
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