Cure SMA Joins SMA Europe and TREAT-NMD for Meeting with EMA Regulators

Leave a Comment / Advocacy, Community Awareness, Front Page News /

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 discuss, support and advance the development of therapies for the treatment of SMA. The workshop will convene key stakeholders—patients, doctors, industry representatives, researchers and regulators—to take stock of the latest

Cure SMA Joins SMA Europe and TREAT-NMD for Meeting with EMA Regulators Read More »

Cure SMA to Host Webinar on the New Drug Application Process

Leave a Comment / Advocacy, Community Awareness, Front Page News, Our Impact /

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 Community Statement on Interim Analysis of CHERISH (Type 2) Trial

Leave a Comment / Clinical Trials, Front Page News /

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 Announce SPINRAZA (nusinersen) Meets Primary Endpoint at Interim Analysis of Phase 3 CHERISH Study

Leave a Comment / Clinical Trials, Front Page News /

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

Biogen and Ionis Pharmaceuticals Announce SPINRAZA (nusinersen) Meets Primary Endpoint at Interim Analysis of Phase 3 CHERISH Study Read More »

AveXis Announces Single-Arm Design for Pivotal Study of AVXS-101

Leave a Comment / Clinical Trials, Front Page News /

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 »

Family Registration for the 2017 Annual SMA Conference is Now Open

Leave a Comment / Conference, Front Page News /

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 Launches Development of STL-182 to Treat SMA

Leave a Comment / Front Page News, Research /

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 »

6th Annual Congressional Dinner Furthers SMA Advocacy Work

Leave a Comment / Advocacy, Front Page News, Our Impact /

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 »

Cure SMA-funded Researcher Sara Custer Publishes Paper

Leave a Comment / Front Page News, Research /

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 »

SMA Community is Granted a Patient Focused Drug Development Meeting with the FDA

Leave a Comment / Advocacy, Community Awareness, Front Page News, Our Impact /

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 »

Scroll to Top