Team Biogen Runs Over 22,000 Miles During SMA Awareness Month

        Team Biogen hit the ground running throughout the month of August in honor of SMA Awareness Month. Together, the team logged 22,059 miles collectively from August 1st to August 31st. Nearly 300 Biogen employees came together, virtually of course, to raise funds and awareness for SMA. They successfully raised close to […]

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Community Spotlight: Braedon’s Newborn Screening Diagnosis

September is Newborn Screening Awareness Month, a chance to talk about the progress we have made in getting spinal muscular atrophy (SMA) added to newborn screening panels and the work still to be done. This month, you will hear about newborn screening milestones, stories, and steps you can take to ensure all states screen newborns

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Biogen Q3 2020 Community Statement: COVID-19 Updates and Latest Milestones

Dear Members of the SMA Community, We continue to work with the healthcare community to help people and families living with SMA get the support they need. As such, we wanted to share Biogen’s latest efforts on the following: We are committed to sharing information that will aid healthcare providers and individuals with SMA in

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Scholar Rock Announces that SRK-015 has Received Rare Pediatric Disease Designation from U.S. FDA for the Treatment of SMA

Scholar Rock, a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation for SRK-015 for the treatment of Spinal Muscular Atrophy (SMA), a progressive, rare genetic disease that

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Community Spotlight: Nicole Stickane Advocates for Newborn Screening in Texas

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

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

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

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

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

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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