Community Spotlight: Nicole Stickane Advocates for Newborn Screening in Texas

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In late July, the Texas Newborn Screening Advisory Committee held a meeting— virtually, of course —to hear an update on the status of statewide newborn screening for spinal muscular atrophy (SMA). State officials shared updates and members of the SMA community shared public comments. Nicole Stickane shared her family’s experience in support of adding SMA […]

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Genentech Releases Evrysdi™ FDA Approval Letter to the SMA Community

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Dear SMA Patient Advocacy Community, As part of our ongoing partnership and following your request to receive updates about the risdiplam clinical development program, we are delighted to share with you a much-anticipated milestone. Today, the U.S. Food and Drug Administration (FDA) has approved risdiplam for the treatment of spinal muscular atrophy (SMA) in adults

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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 Launches New Medical Alert Bracelet Support Program

2 Comments / Front Page News, Support & Care /

Along with funding SMA research and care, Cure SMA provides thousands of individuals and families with vital support and resources that help people navigate life with spinal muscular atrophy (SMA). In addition to our current support programs, Cure SMA is thrilled to launch a brand-new program offered to all affected individuals with SMA to help

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Cure SMA to Meet with FDA for Critical Path Innovation Meeting

1 Comment / Front Page News, Support & Care /

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

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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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Community Spotlight: Avery’s Diagnosis from Newborn Screening

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July 2020 marks the 2-year anniversary of when the U.S. Secretary of Health and Human Services recommended that SMA be added to state newborn screening panels. This month, you’ll hear about newborn screening milestones, stories, and steps you can take to ensure all states screen newborns for SMA. Shannon’s story of her family’s experience with

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Thank You for a Wonderful 2020 Virtual SMA Conference!

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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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