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 12:00pm CST (10:00am PST/11:00am MST/1:00pm EST). Jill Jarecki, chief scientific officer, and Kenneth Hobby, president, will be joined by a two clinical and regulatory experts to discuss the NDA process […]
Cure SMA to Host Webinar on the New Drug Application Process Read More »
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 we take another important step to advance the first potential treatment option for Spinal Muscular Atrophy (SMA). We are happy to continue to provide exciting news about the SPINRAZA (nusinersen)
Biogen and Ionis Community Statement on Interim Analysis of CHERISH (Type 2) Trial Read More »
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 CHERISH, the Phase 3 study evaluating SPINRAZA in later-onset (consistent with Type 2) SMA. The analysis found that children receiving SPINRAZA experienced a highly statistically significant improvement in motor function
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 muscular atrophy (SMA) Type 1 will reflect a single-arm design, using natural history of the disease as a comparator, and enroll approximately 20 patients. This
AveXis Announces Single-Arm Design for Pivotal Study of AVXS-101 Read More »
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 Sunday, July 2, 2017. We are excited to experience Disney together for a second straight year. Disney characters will be at many of our events,
Family Registration for the 2017 Annual SMA Conference is Now Open Read More »
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 Discovery in Neurodegeneration at Brigham and Women’s Hospital. The investigators’ successful research collaboration was supported in part by a series of grants from the National
Spotlight Innovation Launches Development of STL-182 to Treat SMA Read More »
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 are excited to have a number of special guests join us, including Rep. Kevin Brady, Rep. Sean Duffy, Rep. Jeb Hensarling, Rep. Bill Huizenga, Rep.
6th Annual Congressional Dinner Furthers SMA Advocacy Work Read More »
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 in this paper was funded by a basic research grant from Cure SMA. Individuals with SMA don’t produce enough survival motor neuron (SMN) protein, due
Cure SMA-funded Researcher Sara Custer Publishes Paper Read More »
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, individuals and families from throughout our community will have the opportunity to testify directly to the FDA. The PFDD meeting will include testimony on a
SMA Community is Granted a Patient Focused Drug Development Meeting with the FDA Read More »
Back by popular demand, Cure SMA and Ionis Pharmaceuticals are once again teaming up for a holiday card contest! This contest is open to children with spinal muscular atrophy, their siblings, and to children of parents with SMA. Ionis will select three entries to use in their annual holiday card. These winning entries will receive
Cure SMA and Ionis Announce 2016 Holiday Card Contest Read More »
