The organization’s grassroots efforts give thousands of families new hope by securing widespread, state-by-state screening for SMA at birth Within three years of spinal muscular atrophy (SMA) being added to the federally recommended list of diseases to screen for at birth, Cure SMA is celebrating a significant milestone—85 percent of newborns in the U.S. are now […]
Novartis recently announced data that reinforce the transformational benefit of Zolgensma® (onasemnogene abeparvovec), an essential, one-time treatment and the only gene therapy for spinal muscular atrophy (SMA). New late-breaker data from the completed two-copy cohort of the Phase 3 SPR1NT clinical trial demonstrate age-appropriate milestone development in presymptomatic children with SMA without respiratory or nutritional support of
When you first learned that you or your child had SMA, you probably began analyzing your home life, your or your child’s education, and other immediate concerns. In time, you realized that the needs would continue to evolve. How would you pay for medical expenses that insurance did not cover? What types of programs would
Financial Planning with a Disability Read More »
Scholar Rock, a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, announced an oral presentation of TOPAZ Phase 2 trial results by the lead principal investigator, Thomas Crawford, M.D. of Johns Hopkins Medicine. In the TOPAZ trial, treatment with apitegromab in conjunction with nusinersen in patients
Genentech, a member of the Roche Group, today announced new interim data from two studies of Evrysdi® (risdiplam)—JEWELFISH and RAINBOWFISH. Genentech leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics. About JEWELFISH Data Data from JEWELFISH, an ongoing open-label study primarily evaluating the safety of Evrysdi in
Biogen Inc., today announced new research supporting the continued development of an investigational higher dose of SPINRAZA® (nusinersen) and additional data reinforcing the strength of SPINRAZA’s clinical profile in improving the lives of individuals with spinal muscular atrophy (SMA) over the long term. “Intervention with SPINRAZA can meaningfully impact the trajectory of SMA, and we
Biogen Presents New Spinraza Data at the 2021 Virtual SMA Conference Read More »
On the 25th anniversary of the SMA Researcher Meeting, we would like you to learn more about Dr. Burghes and his commitment to the Cure SMA community. Arthur Burghes, Ph.D., is a Professor of Biological Chemistry and Pharmacology at The Ohio State University and an internationally recognized research scientist who has dedicated more than 30 years
Community Spotlight: Dr. Arthur Burghes Read More »
Each year at the Annual SMA Conference, the session on Aquatic Therapy is among the most popular for attendees of all ages. Jennifer Martyn, PT, is one of the leaders of this educational and fun session. Normally this time of year, Jennifer and her staff would begin ordering new gear for the pool, labeling items
Understanding the Benefits of Aqua Therapy Read More »
Cure SMA is excited to share that the platform for the 2021 Virtual SMA Conference and 2021 Virtual SMA Research & Clinical Care Meeting is officially open! Sessions will kick off tomorrow, Monday, June 7th and run through Friday, June 11th, with a few additional social events trickling into the following week. There is still
Ready to Go! Platform Open for 2021 Virtual SMA Conference Events Read More »
In a May 2021 issue of BMC Pediatrics, Cure SMA published a manuscript, titled “Awareness screening and referral patterns among pediatricians in the United States related to early clinical features of spinal muscular atrophy (SMA).” The purpose of the manuscript was to describe the findings of two healthcare provider surveys launched in 2018 and 2019,
