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 »
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 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
Thank You Thank you to everyone who attended the 2026 Annual SMA Conference at Disney’s Yacht & Beach Club Hotel in Orlando, Florida! It was an impactful weekend of opportunities to connect, learn, and have fun. We’re thrilled to share that over 2,700 attendees joined us, hosting the most adults with spinal muscular atrophy
Thank you to everyone who attended the 2026 Annual SMA Conference Read More »
Biogen recently announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to salanersen, an investigational therapy being studied for the treatment of spinal muscular atrophy (SMA). This designation is intended to help speed the development and review of drugs that show early promise in treating serious conditions. Salanersen is an
The Family-Friendly Poster Session is one of the most anticipated events at the Annual SMA Conference! This year’s session will be held on Friday, June 26, from 6:30 pm to 8:30 pm in the Grand Harbor Ballroom at Disney’s Yacht and Beach Club Resort. The Family Friendly Poster Session is an opportunity for families to
Meet the Scientists Behind SMA Research and Discover What’s Coming Next Read More »
As more SMA treatments become available, people living with SMA, their families, and caregivers are asking new questions about how treatments work, whether using more than one treatment could help, and what to consider when thinking about combination therapy. Understanding how SMA treatments work can help individuals and families feel more informed about their options
