Cure SMA is pleased to announce the publication of “Impact of early intervention on motor milestone achievement in spinal muscular atrophy: Insights from Cure SMA survey data” in the April 2026 issue of Neurology and Therapy. Since 2017, Cure SMA has been collecting patient- and caregiver-reported data through the annual Community Update Survey (CUS). The
Winnie is an 11-year-old living with spinal muscular atrophy (SMA) who loves musicals, drawing, and playing sports with her brothers. After her recent surgery, she wrote a book to help other kids get through theirs. If you’ve ever helped a child get ready for surgery, you know the questions that come with it. How long
Meet Winnie, the 11-Year-Old Author Helping Kids Face Surgery Read More »
Today, Cure SMA is proud to launch our year-end campaign: This Gen, Next 10. It’s a chance to look at where this community started, how far we’ve come, and what the next ten years should bring for those impacted by spinal muscular atrophy (SMA). Over the campaign, we’ll revisit the days before treatment existed, when
Cure SMA Launches Their “This Gen Next 10” Campaign Read More »
This week, the unmet health and everyday living needs of adults with spinal muscular atrophy (SMA) took center stage in Washington, DC, in an event organized by Cure SMA. Adults with SMA from across the country participated in the September 27-28 event to discuss the significant challenges and barriers they face that impact their health
Adults with SMA Share Their Unmet Needs and Independence Barriers in Washington D.C. Read More »
From newly opened Phase 3 studies enrolling infants through adults to the first participants already dosed in the U.S. and Japan, Biogen shares an update on where its investigational salanersen program stands today. Read their latest update here.
Biogen Shares Update on the Salanersen Development Program Read More »
In June, SMA researchers and clinicians from around the world gathered in Orlando for the 30th Annual SMA Research & Clinical Care Meeting — to share early findings, compare notes across disciplines, and drive continued progress in clinical care and new treatments for people living with SMA. This year, we’re doing something new: all the
The 2026 Annual SMA Research & Clinical Care Meeting Summaries Are Here! Read More »
Scholar Rock today announced that it has received approval from the U.S. Food and Drug Administration (FDA) for ISEMBYLD™ (apitegromab-mstn) to treat spinal muscular atrophy (SMA) in adults and children 2 years of age and older who are currently receiving a survival motor neuron 2 (SMN2)-targeted treatment. This approval brings the first muscle-targeted treatment to
Scholar Rock Receives Approval for ISEMBYLD™ for the Treatment of SMA Read More »
From a lively “Evryday Café” exhibit hall experience to an advocacy reception featuring conversations on policy and access, Genentech recaps a full week connecting with the SMA community at this year’s Annual SMA Conference. Read their latest community update here.
Genentech Shares Highlights from an Unforgettable Week at the Annual SMA Conference Read More »
From their time at the Cure SMA Annual Conference, to their SMAshing My Limits partnership with Becoming rentABLE, opening up accessible beach houses, treehouses, and yurts for family travel, to adaptive sports and recreation panels, Novartis recaps a busy summer of community connection. Read their latest community update here.
Novartis Highlights Accessible Travel Partnership in Summer Community Update Read More »
As increased treatment options have helped more people with spinal muscular atrophy (SMA) live longer, healthier lives, more young adults are navigating the transition to adult care. This means moving from the pediatric care team that, in many cases, has known them since childhood to adult healthcare systems where they are meeting providers for the
