Biogen, Inc., today announced new results from NURTURE, the longest study of pre-symptomatic patients with spinal muscular atrophy (SMA), which is transforming expectations of early treatment with SPINRAZA (nusinersen). In infants genetically diagnosed with SMA, new data demonstrate that early and sustained treatment with SPINRAZA for up to 4.8 years enabled unprecedented […]
Cure SMA is pleased to announce that the 2020 Virtual SMA Conference booklet is now available for download. The booklet includes important details on the Virtual SMA Conference, including: A full agenda with event descriptions and speaker bios for the 16 virtual topical workshops. Don’t forget to register for each individual event that you are
2020 Virtual SMA Conference Booklet is Now Available Online Read More »
In the midst of the ongoing Coronavirus (COVID-19) global pandemic, Cure SMA is excited to announce its new funding for the Pediatric Neuromuscular Clinical Research Network (PNCRN). In 2020, Cure SMA will provide $1.2 million to this Network of highly skilled clinical trial investigators, clinical evaluators, clinical coordinators, statisticians, and data management personnel. The PNCRN
Cure SMA to Fund Network of Prominent SMA Clinical Research and Treatment Centers Read More »
Cure SMA is proud to share with the SMA community that it has published a paper in the Orphanet Journal of Rare Diseases, titled ‘The SMA Clinical Trial Readiness Program: Creation and Evaluation of a Program to Enhance SMA Trial Readiness in the United States.’ The Cure SMA Clinical Trial Readiness Program is
Cure SMA Publishes Article on SMA Clinical Trial Readiness Program in the U.S. Read More »
Cure SMA has released the agenda and registration details for the 2020 Virtual SMA Conference, which will be held from Monday, June 8th – Friday, June 12th. We greatly look forward to virtually uniting as a community in just a few short weeks! It is our #SMAConferenceAtHome. Check out the Virtual SMA Conference Booklet for
Registration Now Open for the 2020 Virtual SMA Conference Read More »
New SPINRAZA® (nusinersen) Data Reinforce Sustained Efficacy and Longer-Term Safety Across Broad Range of SMA Patients Biogen Inc. recently announced additional data from the SPINRAZA (nusinersen) clinical development program that further demonstrate the sustained efficacy and longer-term safety of SPINRAZA in a broad range of patients with spinal muscular atrophy (SMA). These new data were
Biogen Shares SPINRAZA Research Update Read More »
Cure SMA has been hosting the Annual SMA Conference since 1988, bringing together leading researchers, clinicians, affected individuals, and families living with SMA. The weekend has traditionally been filled with a wide variety of opportunities to learn about the latest in treatments, research, advocacy, care, and support. But most of all, it was a time
Cure SMA Announces a 2020 Virtual SMA Conference – United, At Home Read More »
For the past two years, Cure SMA’s Summit of Strength program has brought together thousands of SMA community members for the opportunity to network and learn about the latest advances in treatment, care, advocacy, and support. Considering event cancellations across the country due to the ongoing pandemic, Cure SMA is now offer these resources through
Cure SMA Announces Summit of Strength Virtual Webinar Series Read More »
Dear Members of the SMA Community, In light of the COVID-19 pandemic and the burden it has placed on our healthcare system, Biogen is working with health systems, institutions, physicians, patients and their families to navigate potential SPINRAZA® (nusinersen) dosing delays related to COVID-19. The health and safety of patients is our primary concern, and
Scholar Rock recently reported financial results for the first quarter ending March 31, 2020, highlighting progress and upcoming milestones for its pipeline programs—include the SRK-015 program for spinal muscular atrophy (SMA). Scholar Rock plans to report interim efficacy, safety, pharmacokinetic (PK), and pharmacodynamic (PD) results in the fourth quarter of 2020 for patients across the
Update from Scholar Rock on TOPAZ Phase 2 Clinical Trial of SRK-015 in Patients with SMA Read More »
