Industry Updates
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.
Read More ›Biogen Shares DEVOTE and RESPOND Study Updates
Biogen recently shared that they have completed enrollment in the global DEVOTE and RESPOND clinical studies. Check out the full SMA Community Statement here.
Read More ›Genentech Shares 1-Year Primary Analysis from RAINBOWFISH
Last week, Genentech, a member of the Roche Group, presented positive results from the primary analysis of the ongoing RAINBOWFISH study assessing the efficacy and […]
Read More ›Novartis Gene Therapies SMA Community Update
Dear SMA Community, It was a pleasure to be able to join so many of you at the Cure SMA Annual Conference in Orlando this […]
Read More ›SMA Highlights and Updates from Biogen
Dear SMA Community, As we reflect on SMA Awareness Month, we take this opportunity to celebrate all those who are a part of the SMA […]
Read More ›NMD Pharma Initiates Phase II Trial for SMA
NMD Pharma, a clinical-stage biotech company leading the development of novel first-in-class therapies for severe neuromuscular disorders, recently announced that the first SMA patient has […]
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