Industry Updates
Majority of Children with SMA Treated with Genentech’s Evrysdi Are Able to Sit, Stand and Walk Independently, Two-Year Data Demonstrate
Positive data confirm Evrysdi efficacy and safety in children first treated pre-symptomatically before 6 weeks of age, with most achieving motor milestones similar to children […]
New Higher Dose Nusinersen Efficacy and Safety Data Presented at World Muscle Society Congress, Highlight Potential to Maximize Benefit of Nusinersen in SMA
Findings from Part B and Part C of the DEVOTE study support the clinical benefits of a higher dose regimen of nusinersen (50/28 mg) in […]
Scholar Rock Announces Apitegromab Meets Primary Endpoint in Phase 3 SAPPHIRE Study in Patients with Spinal Muscular Atrophy (SMA)
Today, Scholar Rock announced plans to submit a U.S. Biologics License Application (BLA) and a European Union marketing authorisation application (MAA) in Q1 2025. Scholar […]
Biogen Announces Positive Topline Results from Study of Higher Dose Regimen of Nusinersen, Showing Significant Benefit in Treatment of SMA
The positive study demonstrates the potential for investigational higher dose nusinersen regimen to advance the treatment of SMA. Biogen plans to submit for regulatory approval […]
SMA Highlights: Updates from Biogen
On July 12, 2024, Biogen shared highlights and updates from the 2024 Annual SMA Conference, including their collaboration with Delta Flight Products and their sponsorship […]
Five-Year Data for Genentech’s Evrysdi Show the Majority of Treated Children With a Severe Form of SMA Achieved or Maintained the Ability to Sit, Stand or Walk
Summary of Data Presented After 5 years of treatment, 91% of children were alive – without treatment, children with Type 1 SMA would not be […]
Novartis Gene Therapies Releases Spring SMA Community Letter
Novartis Gene Therapies recently released an update to the SMA community related to clinical trials and long term follow up studies of Zolgensma, in addition […]
Genentech Releases SMA Community Letter Recapping Spring Activities
On May 3, 2024, Genentech released an update to the SMA community related to Evrysdi® (risdiplam) and Genentech’s activities in service of the SMA community. […]
Biogen Releases SMA Community Letter
On April 19, 2024, Biogen released an update to the SMA community sharing recent data presentations from Spring conferences and latest updates from their SPINRAZA® […]
Apitegromab Linked to Improved Outcomes for Those with Spinal Muscular Atrophy
The study included 58 individuals with SMA types 2 and 3 who were treated with IV apitegromab for 52 weeks. At 36 months, treatment resulted […]