Author name: Leslie Humbel

Novartis Announces Lift of Partial Clinical Trial Hold, Plans to Initiate a New Study of Intrathecal Zolgensma in Older Patients with SMA

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Novartis announced that the U.S. Food and Drug Administration (FDA) has determined that OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may proceed, thereby lifting the partial clinical trial hold initiated in October 2019. The decision to lift the hold was based on data from Novartis’ comprehensive nonclinical toxicology study in non-human …

Novartis Announces Lift of Partial Clinical Trial Hold, Plans to Initiate a New Study of Intrathecal Zolgensma in Older Patients with SMA Read More »

Data for Genentech’s Evrysdi Published in New England Journal of Medicine Shows Significant Improvement in Survival and Motor Milestones in Babies with Type 1 SMA

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Data from Genentech, a member of the Roche Group, recently announced that the New England Journal of Medicine (NEJM) has published data from FIREFISH Part 2, a pivotal global study evaluating the efficacy and safety of Evrysdi® (risdiplam) in babies aged 1-7 months old with symptomatic Type 1 spinal muscular atrophy (SMA). The study met its primary endpoint …

Data for Genentech’s Evrysdi Published in New England Journal of Medicine Shows Significant Improvement in Survival and Motor Milestones in Babies with Type 1 SMA Read More »

In Just Three Years, Cure SMA Marks Nationwide Milestone for 85% of U.S. Babies Now Screened at Birth for SMA

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The organization’s grassroots efforts give thousands of families new hope by securing widespread, state-by-state screening for SMA at birth Within three years of spinal muscular atrophy (SMA) being added to the federally recommended list of diseases to screen for at birth, Cure SMA is celebrating a significant milestone—85 percent of newborns in the U.S. are now …

In Just Three Years, Cure SMA Marks Nationwide Milestone for 85% of U.S. Babies Now Screened at Birth for SMA Read More »

Novartis Data Demonstrate Age-Appropriate Development When Zolgensma Is Used Presymptomatically and Rapid, Clinically Meaningful Efficacy in Symptomatic Children

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Novartis recently announced data that reinforce the transformational benefit of Zolgensma® (onasemnogene abeparvovec), an essential, one-time treatment and the only gene therapy for spinal muscular atrophy (SMA). New late-breaker data from the completed two-copy cohort of the Phase 3 SPR1NT clinical trial demonstrate age-appropriate milestone development in presymptomatic children with SMA without respiratory or nutritional support of …

Novartis Data Demonstrate Age-Appropriate Development When Zolgensma Is Used Presymptomatically and Rapid, Clinically Meaningful Efficacy in Symptomatic Children Read More »

Scholar Rock Presents TOPAZ Phase 2 Data Showing the Transformative Potential of Apitegromab in Patients with Type 2 and 3 SMA

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Scholar Rock, a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, announced an oral presentation of TOPAZ Phase 2 trial results by the lead principal investigator, Thomas Crawford, M.D. of Johns Hopkins Medicine. In the TOPAZ trial, treatment with apitegromab in conjunction with nusinersen in patients …

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New Genentech Data for Evrysdi Show Improved Motor Function in Pre-Symptomatic Babies After One Year and Confirm Safety Profile in Previously Treated People with SMA

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Genentech, a member of the Roche Group, today announced new interim data from two studies of Evrysdi® (risdiplam)—JEWELFISH and RAINBOWFISH. Genentech leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics. About JEWELFISH Data Data from JEWELFISH, an ongoing open-label study primarily evaluating the safety of Evrysdi in …

New Genentech Data for Evrysdi Show Improved Motor Function in Pre-Symptomatic Babies After One Year and Confirm Safety Profile in Previously Treated People with SMA Read More »

Ready to Go! Platform Open for 2021 Virtual SMA Conference Events

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Cure SMA is excited to share that the platform for the 2021 Virtual SMA Conference and 2021 Virtual SMA Research & Clinical Care Meeting is officially open! Sessions will kick off tomorrow, Monday, June 7th and run through Friday, June 11th, with a few additional social events trickling into the following week. There is still …

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Cure SMA Publishes Manuscript on SMA Awareness, Screening, and Referral Patterns Amongst Pediatricians

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In a May 2021 issue of BMC Pediatrics, Cure SMA published a manuscript, titled “Awareness screening and referral patterns among pediatricians in the United States related to early clinical features of spinal muscular atrophy (SMA).” The purpose of the manuscript was to describe the findings of two healthcare provider surveys launched in 2018 and 2019, …

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Cure SMA Announces 2022 Annual SMA Conference

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Cure SMA is excited to announce that we will reunite the SMA community in-person for the 2022 Annual SMA Conference in Anaheim, CA–Thursday, June 16 through Sunday, June 19, 2022. If the unexpected circumstances surrounding the pandemic have shown us anything, it is how impactful this one week of the year is for individuals, families, …

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Community Statement: Quarterly Update from Novartis Gene Therapies

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Dear Members of the SMA Community, Novartis Gene Therapies is committed to working with our partners in the SMA community to make a difference in the diagnosis, treatment, and care of those with SMA.  Through our engagement with you directly, and through our collaborations with Cure SMA, together we can make a difference. We are …

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