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 […]

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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 »

Biogen Presents New Spinraza Data at the 2021 Virtual SMA Conference

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Biogen Inc., today announced new research supporting the continued development of an investigational higher dose of SPINRAZA® (nusinersen) and additional data reinforcing the strength of SPINRAZA’s clinical profile in improving the lives of individuals with spinal muscular atrophy (SMA) over the long term. “Intervention with SPINRAZA can meaningfully impact the trajectory of SMA, and we

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Community Spotlight: Dr. Arthur Burghes

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On the 25th anniversary of the SMA Researcher Meeting, we would like you to learn more about Dr. Burghes and his commitment to the Cure SMA community. Arthur Burghes, Ph.D., is a Professor of Biological Chemistry and Pharmacology at The Ohio State University and an internationally recognized research scientist who has dedicated more than 30 years

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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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