News
May 28, 2019
Cure SMA to Host Adult Lounge and Expanded Adult Social at Annual Conference Thanks to Biogen and the Dhont Family Foundation
Cure SMA will be offering two great programming opportunities for adults (18 and over) with SMA attending the 2019 Annual SMA Conference next month – an adult lounge and two adult social events.
Thanks to a generous grant funded by Biogen, Cure SMA will be providing an exclusive...
Topics: Conference, Front Page 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...
Topics: Research, Front Page News
May 24, 2019
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...
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, 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,...
Topics: Clinical Trials, Research, Front Page News
May 21, 2019
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...
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...
Topics: Our Impact, Research, Front Page News
May 16, 2019
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...
Topics: Clinical Trials, Research, Front Page News
May 16, 2019
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...
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 Inc. today announced that The National Institute for Health and Care Excellence (NICE) in the United Kingdom has recommended funding for SPINRAZA...
Topics: Research, Front Page News
May 14, 2019
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...
Topics: Our Impact, Research, Front Page News