Cure SMA Convenes Working Group to Discuss Biomarkers for SMA

Leave a Comment / Front Page News, Research /

The advent of three approved SMN-upregulating therapeutics for spinal muscular atrophy has dramatically changed the natural history of the disease. There are, however, many uncertainties regarding timing of treatment initiation, response to treatment, and long-term outcomes.  The identification of biomarkers, medical indicators that can inform us as to disease status, treatment response, and/or provide information […]

Cure SMA Convenes Working Group to Discuss Biomarkers for SMA Read More »

Thank You for Another Fantastic Virtual SMA Conference!

Leave a Comment / Conference, Front Page News /

Cure SMA would like to thank everyone who attended the recent 2021 Virtual SMA Conference and 2021 Virtual SMA Research & Clinical Care Meeting. It was an impactful week of workshops, networking, and SMA community engagement. This year’s virtual conference week—the second virtual conference during these pandemic times—hosted nearly 2,500 registrants from over 65 countries. Cure SMA offered

Thank You for Another Fantastic Virtual SMA Conference! Read More »

In Just Three Years, Cure SMA Marks Nationwide Milestone for 85% of U.S. Babies Now Screened at Birth for SMA

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

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

In Just Three Years, Cure SMA Marks Nationwide Milestone for 85% of U.S. Babies Now Screened at Birth for SMA Read More »

Novartis Data Demonstrate Age-Appropriate Development When Zolgensma Is Used Presymptomatically and Rapid, Clinically Meaningful Efficacy in Symptomatic Children

Leave a Comment / Clinical Trials, Front Page News, Industry Updates, Research /

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

Novartis Data Demonstrate Age-Appropriate Development When Zolgensma Is Used Presymptomatically and Rapid, Clinically Meaningful Efficacy in Symptomatic Children Read More »

Scholar Rock Presents TOPAZ Phase 2 Data Showing the Transformative Potential of Apitegromab in Patients with Type 2 and 3 SMA

1 Comment / Conference, Front Page News, Industry Updates, Research /

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

Scholar Rock Presents TOPAZ Phase 2 Data Showing the Transformative Potential of Apitegromab in Patients with Type 2 and 3 SMA Read More »

New Genentech Data for Evrysdi Show Improved Motor Function in Pre-Symptomatic Babies After One Year and Confirm Safety Profile in Previously Treated People with SMA

1 Comment / Conference, Front Page News, Industry Updates, Research /

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

New Genentech Data for Evrysdi Show Improved Motor Function in Pre-Symptomatic Babies After One Year and Confirm Safety Profile in Previously Treated People with SMA Read More »

Biogen Presents New Spinraza Data at the 2021 Virtual SMA Conference

1 Comment / Front Page News, Industry Updates, Research /

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 »

Community Spotlight: Dr. Arthur Burghes

Leave a Comment / Community Awareness, Community Spotlight, Conference /

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 »

Scroll to Top