Biogen Inc., today announced, it plans to initiate a global Phase 4 clinical study, RESPOND, to examine the clinical benefit and assess the safety of SPINRAZA (nusinersen) in infants and children with spinal muscular atrophy (SMA) who still have unmet clinical needs following treatment with gene therapy Zolgensma (onasemnogene abeparvovec). People with SMA do not produce enough survival […]
July 2020 marks the 2-year anniversary of when the U.S. Secretary of Health and Human Services recommended that SMA be added to state newborn screening panels. This month, you’ll hear about newborn screening milestones, stories, and steps you can take to ensure all states screen newborns for SMA. Shannon’s story of her family’s experience with
Community Spotlight: Avery’s Diagnosis from Newborn Screening Read More »
Ben Lou is a 17-year-old from San Diego who is fluent in both English and Chinese. He is a math whiz and fascinated by science. Ben also has had total body weakness from birth and, at the age of 1 year, was diagnosed with spinal muscular atrophy (SMA). His experience, like many others, led to
Community Spotlight: Ben Lou’s Formula for Life Read More »
Cure SMA would like to thank everyone who attended last week’s 2020 Virtual SMA Conference! It was an impactful week of online workshops, networking, and community. We are happy to announce that there were more than 6,600 participants who attended the 28 offered live events. Although we were unable to come together in-person, it was
Thank You for a Wonderful 2020 Virtual SMA Conference! Read More »
Genentech, a member of the Roche Group, shared two-year data from Part 1 of its pivotal SUNFISH trial in people aged 2-25 years with Type 2 or 3 spinal muscular atrophy (SMA). This was presented at the 2020 Virtual SMA Research & Clinical Care Meeting. The results of an exploratory efficacy analysis
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 »
