News

May 24, 2019

AveXis and Cure SMA to Co-Host Webinar on the FDA's Approval of Zolgensma

On Wednesday, June 12th, at 10am CDT (8am PDT/9am MDT/11am EDT) AveXis, a Novartis company, and Cure SMA will co-host a webinar on the FDA approval of Zolgensma. This webinar will include information about the approval, dosing sites, future IT clinical trials, and more. The webinar will also include...

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Topics: Research, Front Page News

May 24, 2019

AveXis Issues Community Statement on Zolgensma Approval

AveXis Issues Community Statement on Zolgensma Approval

Dear SMA Community,

We have exciting news to share with you — a major milestone has been reached in the fight against spinal muscular atrophy (SMA)! The US Food and Drug Administration (FDA) has approved ZOLGENSMA® (onasemnogene abeparvovec-xioi) for the treatment of children...

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Topics: Clinical Trials, Research, Front Page News

May 24, 2019

AveXis Receives FDA Approval of Zolgensma, a Gene Therapy, for Spinal Muscular Atrophy for Patients Under Two Years of Age

AveXis Receives FDA Approval of Zolgensma, a Gene Therapy, for Spinal Muscular Atrophy for Patients Under Two Years of Age

AveXis, Inc., a Novartis company, today announced that they have received FDA approval for Zolgensma (formerly AVXS-101), a gene therapy that replaces the survival motor neuron 1 (SMN1) gene,...

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Topics: Clinical Trials, Research, Front Page News

May 21, 2019

Cure SMA Awards $150,000 Grant to Krysta Engel, PhD, University of Colorado

Cure SMA Awards $150,000 Grant to Krysta Engel, PhD, University of Colorado

Audrey Lewis founded Families of SMA, now Cure SMA, 34 years ago. Audrey recognized early on the importance of attracting new and talented researchers to SMA, with the hope that they would commit their careers to developing a treatment and cure for SMA.

Cure SMA honors Audrey’s...

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Topics: Our Impact, Research, Front Page News

May 17, 2019

Cure SMA Attends the 71st Annual American Academy of Neurology (AAN) Meeting in Philadelphia

The American Academy of Neurology (AAN) 2019 Annual meeting wrapped up in Philadelphia, Pennsylvania on May 10th, 2019. The AAN Annual meeting is one of the world’s largest professional meeting of neurologists on cutting-edge science, research, and clinical updates. Over 14,000 neurology professionals and meeting attendees were in...

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Topics: Our Impact, Research, Front Page News

May 16, 2019

Novartis Issues Community Statement on Branaplam Clinical Study

Novartis Issues Community Statement on Branaplam Clinical Study

Dear SMA Community,

We are writing to inform you that enrollment to the ongoing branaplam clinical study is now closed.

Branaplam is currently being assessed for safety and efficacy in a Phase 1/2 clinical study in Type 1 infantile-onset SMA. We are pleased to announce...

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Topics: Clinical Trials, Research, Front Page News

May 16, 2019

Team Biogen Runs from Chicago to Madison, Raising Over $12,000 for Cure SMA

Team Biogen Runs from Chicago to Madison, Raising Over $12,000 for Cure SMA

On Friday, May 17th to Saturday, May 18th, Team Biogen will hit the ground running in the 2019 Reebok Ragnar Great Midwest, a 14-person relay race. The team's goal is to raise funds and awareness for the SMA community.

Team Biogen has already raised over $12,000 for Team Cure SMA while...

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Topics: Events & Fundraising, Front Page News

May 15, 2019

Biogen’s SPINRAZA® (nusinersen) Receives Positive Recommendation from NICE for Funding in the United Kingdom for the Treatment of Infants, Children and Adults with Spinal Muscular Atrophy

Biogen’s SPINRAZA® (nusinersen) Receives Positive Recommendation from NICE for Funding in the United Kingdom for the Treatment of Infants, Children and Adults with Spinal Muscular Atrophy

Biogen Inc. today announced that The National Institute for Health and Care Excellence (NICE) in the United Kingdom has recommended funding for SPINRAZA...

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Topics: Research, Front Page News

May 14, 2019

Cure SMA Awards $150,000 Grant to Sibylle Jablonka, PhD, University of Würzburg, Germany

Cure SMA Awards $150,000 Grant to Sibylle Jablonka, PhD, University of Würzburg, Germany

Cure SMA has awarded a $150,000 research grant to Sibylle Jablonka, PhD at University of Wurzburg, for her project, “Modulation of calcium channels in mouse models for spinal muscular atrophy (SMA)”.

Dysregulated cellular mechanisms, such as neurotransmission, are complex...

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Topics: Our Impact, Research, Front Page News

May 14, 2019

Cure SMA Publishes Journal Article on Benefit-Risk Preferences in SMA

Your voice has been heard! 

Cure SMA has published a paper, titled, Evaluating Benefit-Risk Decision-Making in Spinal Muscular Atrophy: A First-Ever Study to Assess Risk Tolerance in the SMA Patient Community in the Journal of...

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Topics: Support & Care, Research, Front Page News

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