September 2020

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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Community Spotlight: Xavier and Rose’s SMA Diagnosis

Leave a Comment / Advocacy, Community Spotlight, Front Page News /

Carrie Menke is a South Dakota native who resides in Sioux Falls with her husband Tony and their four children — Xavier, Colette, Avila, and Rose. Carrie is one of the many advocates across the country who is supporting efforts to have spinal muscular atrophy (SMA) added to her state’s newborn screening panel. Her passion

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

Leave a Comment / Clinical Trials, Front Page News /

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

Leave a Comment / Events & Fundraising, Front Page News /

        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

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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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