For Cure SMA, 2018 has been a year of great progress. As we continue to make strides into 2019, it is important to take a moment to look back at all our successes. Thank you to all our families and advocates for your support and continued efforts to cure SMA. We wish you all a […]
Advocacy Year-in-Review 2018 Read More »
Bigoen has provided the below community statement on Spinraza. Dear Members of the SMA community, As we approach the two-year anniversary of the U.S. Food and Drug Administration’s (FDA) approval of SPINRAZA® (nusinersen), we want to thank you for your ongoing support. We share your commitment to advancing research to improve the lives of those
Biogen Issues Q4 Community Statement on Spinraza Read More »
AveXis has provided the following community statement on the BLA submission and acceptance for their investigational therapy, AVXS-101 – now known as ZOLGENSMA®. Dear SMA Community, We are providing this update to the SMA community in the U.S. at the request of Cure SMA. Following our recent October update about our Biologics License Application (BLA)
AveXis Issues Community Statement on BLA Acceptance Read More »
The SMA community has multiple victories to celebrate as the year comes to an end. Thank you to all our amazing families and advocates who have and continue to make this progress possible. Virginia Adopts SMA Newborn Screening Last month, Virginia’s Newborn Screening Advisory Committee adopted SMA to their Newborn Screening Panel. The state government
The fall 2018 issue of Compass is now available online. This issue of Compass covers the SMA Care Center Network and its goal of developing an evidence-based standard of care to improve the lives of people with SMA. SMA Care Center Network In October, Cure SMA launched the SMA Care Center Network, a collection of specialized
Fall 2018 Issue of Compass Now Available Read More »
Novartis today announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s Biologics License Application (BLA) for AVXS-101, now known as ZOLGENSMA® (onasemnogene abeparvovec-xxxx)1, an investigational gene replacement therapy for the treatment of spinal muscular atrophy (SMA) Type 1. ZOLGENSMA is designed to address the genetic root cause of SMA Type 1,
Novartis Announces FDA Filing Acceptance and Priority Review for AVXS-101 Read More »
On Wednesday, December 12th, at 12:00pm CST (10:00am PST/11:00am MST/1:00pm EST) Cure SMA will hold a webinar updating the community on the latest information on SMA treatments and trials. Topics covered will include: AveXis’s BLA filing with the FDA Status of access to Spinraza Ongoing and upcoming SMA clinical trials As with previous discussions, the
Register for Cure SMA’s Webinar on SMA Treatments and Clinical Trials 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, today announced the publication, “Specific Inhibition of Myostatin Activation is Beneficial in Mouse Models of SMA Therapy” in the peer-reviewed journal Human Molecular Genetics. The publication details preclinical studies demonstrating that a highly
Cure SMA today launches SMArt Moves, a new disease awareness and educational campaign to empower parents, pediatricians and other healthcare professionals to swiftly recognize and diagnose the early signs of motor delays. This allows infants and children diagnosed with SMA (spinal muscular atrophy) to receive life-changing—and in some cases, life-saving—treatment. Infants and toddlers diagnosed with
Cure SMA Launches “SMArt Moves” for Early Diagnosis and Treatment Read More »
The 8th Annual “Hope on the Hill” Congressional Dinner will be held on Tuesday, November 27, bringing together SMA families, government officials, and industry leaders to look forward to the next stages for our community. Through this event, we hope to build momentum in the Washington D.C. area to increase awareness of our mutual goals
