Industry Updates

Apitegromab Linked to Improved Outcomes for Those with Spinal Muscular Atrophy

March 12, 2024
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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 […]

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New Biomarker Data Add Further Evidence Supporting the Potential Benefit of SPINRAZA® (nusinersen) in Infants and Toddlers with Unmet Clinical Needs after Gene Therapy

March 6, 2024
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  New data from the RESPOND study show that neurofilament levels, an indicator of neurodegeneration, were reduced in nearly all study participants treated with SPINRAZA […]

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Novartis Presents New Data on Safety and Efficacy of Zolgensma, Including Maintained and Improved Motor Milestones in Older and Heavier Children with SMA

March 4, 2024
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  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 […]

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Novartis Gene Therapies Releases SMA Community Statement

December 11, 2023
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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 […]

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Genentech Releases SMA Community Letter with Evrysdi (risdiplam) Updates

December 7, 2023
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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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Biogen Shares DEVOTE and RESPOND Study Updates

October 26, 2023
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Biogen recently shared that they have completed enrollment in the global DEVOTE and RESPOND clinical studies. Check out the full SMA Community Statement here.

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Genentech Shares 1-Year Primary Analysis from RAINBOWFISH

October 9, 2023
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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 […]

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Novartis Gene Therapies SMA Community Update

October 5, 2023
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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 […]

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SMA Highlights and Updates from Biogen

October 2, 2023
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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 […]

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NMD Pharma Initiates Phase II Trial for SMA

September 26, 2023
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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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