Author name: Leslie Humbel

Community Spotlight: AJ Brockman of Brewhouse Gallery and Kelsey City Brewing Company

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October is National Disability Employment Awareness Month, with an official theme of Increasing Access and Opportunity. Throughout the month, we will be posting stories about adults with SMA in the workforce and are pleased to share this story about AJ Brockman, 32, of Palm Beach Gardens, FL. AJ Brockman says he wears many hats in life. No […]

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Genentech Presents New 2-Year Data for Evrysdi (risdiplam) in Infants with Type 1 SMA

Leave a Comment / Clinical Trials, Front Page News /

Genentech, a member of the Roche Group, today announced new 2-year data from Part 1 of the pivotal FIREFISH study of Evrysdi™ (risdiplam) in infants aged 2-7 months with symptomatic Type 1 spinal muscular atrophy (SMA). The 2-year results in infants treated with the therapeutic dose of Evrysdi (17/21) showed that they continued to improve

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Novartis Issues Community Statement with Update on AVXS-101 Intrathecal Clinical Development Program

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Dear SMA Community, We recently received important feedback from the U.S. Food and Drug Administration (FDA) following their review of data from the STRONG study of AVXS-101 intrathecal (IT) formulation in older patients with spinal muscular atrophy (SMA) and we’d like to share an update with you.  (Please see the press release issue by Novartis

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Genentech Receives FDA Approval of Evrysdi (risdiplam) for the Treatment of SMA

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Genentech, a member of the Roche Group, today announced that it has received approval from the U.S. Food and Drug Administration (FDA) for Evrysdi (risdiplam) to treat adults and children 2 months and older with spinal muscular atrophy (SMA). Evrysdi showed clinically meaningful improvements in motor function across two clinical trials in people with varying ages and levels

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Cure SMA to Meet with FDA for Critical Path Innovation Meeting

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We are excited to share with the SMA community that the U.S. Food and Drug Administration (FDA) has granted Cure SMA a Critical Path Innovation Meeting (CPIM). This means that key leaders at the FDA will have the opportunity to communicate with clinical investigators, members of academia, industry, scientific groups, and patients to improve efficiency

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Biogen Plans to Initiate Phase 4 Study Evaluating Benefit of Spinraza in Patients Treated with Zolgensma

Leave a Comment / Clinical Trials, Front Page News /

Biogen Inc., today announced, it plans to initiate a global Phase 4 clinical study, RESPOND, to examine the clinical benefit and assess the safety of SPINRAZA (nusinersen) in infants and children with spinal muscular atrophy (SMA) who still have unmet clinical needs following treatment with gene therapy Zolgensma (onasemnogene abeparvovec). People with SMA do not produce enough survival

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Thank You for a Wonderful 2020 Virtual SMA Conference!

3 Comments / Conference, Front Page News /

Cure SMA would like to thank everyone who attended last week’s 2020 Virtual SMA Conference! It was an impactful week of online workshops, networking, and community. We are happy to announce that there were more than 6,600 participants who attended the 28 offered live events. Although we were unable to come together in-person, it was

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Genentech Announces 2-Year Risdiplam Data From SUNFISH and New Data From JEWELFISH in Infants, Children and Adults with SMA

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      Genentech, a member of the Roche Group, shared two-year data from Part 1 of its pivotal SUNFISH trial in people aged 2-25 years with Type 2 or 3 spinal muscular atrophy (SMA). This was presented at the 2020 Virtual SMA Research & Clinical Care Meeting. The results of an exploratory efficacy analysis

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Biogen Shares Results from Landmark NURTURE Study of Pre-Symptomatic SMA Patients Treated with Spinraza

1 Comment / Conference, Research /

      Biogen, Inc., today announced new results from NURTURE, the longest study of pre-symptomatic patients with spinal muscular atrophy (SMA), which is transforming expectations of early treatment with SPINRAZA (nusinersen). In infants genetically diagnosed with SMA, new data demonstrate that early and sustained treatment with SPINRAZA for up to 4.8 years enabled unprecedented

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