Research
Biogen Announces New NURTURE Data at the 2019 Annual SMA Conference
Biogen today announced new results from the NURTURE study, adding data to the longest study of spinal muscular atrophy (SMA) in pre-symptomatic infants (n=25). These […]
Read More ›Updated SMA Drug Pipeline Now Available
We’ve recently released an update to the SMA drug pipeline. This latest version includes: 23 active programs, including two approved therapies. 15 pharmaceutical partners. 6 […]
Read More ›Cytokinetics Releases New Data from Reldesemtiv Clinical Study at the 2019 Annual SMA Conference
Cytokinetics, Incorporated, today announced that data from two preclinical studies of reldesemtiv were presented at the 2019 Annual Cure SMA Conference in Anaheim, CA, showing […]
Read More ›Family Friendly Research Poster Session Presenters Announced
The Family Friendly Research Poster Session will be held on Saturday, June 29th from 6:30pm to 8:00pm at the 2019 Annual SMA Conference. The Family […]
Read More ›Biogen Issues Q2 Community Statement on Spinraza
Dear members of the SMA community, We are grateful for the opportunity to continue to help support individuals with spinal muscular atrophy (SMA), their caregivers […]
Read More ›Scholar Rock Announces Positive Final Results from Phase 1 Clinical Trial of SRK-015 in Healthy Volunteers
Scholar Rock today announced positive final top-line results from the Phase 1 clinical trial of its product candidate, SRK-015, a highly specific inhibitor of myostatin […]
Read More ›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 […]
Read More ›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 […]
Read More ›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 […]
Read More ›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 […]
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