Industry Updates
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 […]
Read More ›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 […]
Read More ›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 […]
Read More ›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. […]
Read More ›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® […]
Read More ›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 […]
Read More ›New Biomarker Data Add Further Evidence Supporting the Potential Benefit of SPINRAZA® (nusinersen) in Infants and Toddlers with Unmet Clinical Needs after Gene Therapy
New data from the RESPOND study show that neurofilament levels, an indicator of neurodegeneration, were reduced in nearly all study participants treated with SPINRAZA […]
Read More ›Novartis Presents New Data on Safety and Efficacy of Zolgensma, Including Maintained and Improved Motor Milestones in Older and Heavier Children with SMA
The SMART study supplements a growing body of evidence on the use of Zolgensma in a patient population older and heavier (1.5 – 9.1 […]
Read More ›Novartis Gene Therapies Releases SMA Community Statement
This week, Novartis Gene Therapies released an SMA community statement detailing the progress on enrollment for both STEER and STRENGTH clinical trials, their investigational intrathecal […]
Read More ›Genentech Releases SMA Community Letter with Evrysdi (risdiplam) Updates
Our partners at Genentech recently provided an SMA Community Letter regarding Evrysdi® (risdiplam) and their clinical development program. Visit this link to learn more.
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