Clinical Trials
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 ›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 […]
Read More ›Scholar Rock Issues Community Statement on the Initiation of a New Clinical Trial for SRK-015
The below statement is a community update from Scholar Rock on SRK-015. Dear SMA Community, Scholar Rock is dedicated to developing novel medicines to improve […]
Read More ›Genentech Presents Data from the Risdiplam Pivotal FIREFISH and SUNFISH Studies in Spinal Muscular Atrophy at the AAN Annual Meeting
Genentech, a member of the Roche Group, recently announced new data at the 71st American Academy of Neurology (AAN) Annual Meeting from the dose-finding Part […]
Read More ›AveXis Presents Data at AAN Annual Meeting Demonstrating Efficacy of Zolgensma in Broad Spectrum of Spinal Muscular Atrophy (SMA) Patients
AveXis, a Novartis company, recently announced interim data from ongoing trials of the investigational product Zolgensma® (onasemnogene abeparvovec-xioi; AVXS-101) that showed positive results across a […]
Read More ›Biogen to Present Data at AAN Highlighting Spinraza and Emerging Biomarkers for SMA
Biogen today announced it will present new data focusing on SMA at the 71st annual meeting of the American Academy of Neurology (AAN) in Philadelphia, […]
Read More ›Genentech Presents New Data at the 2019 AAN Annual Meeting
Genentech, a member of the Roche Group, announced today that new data will be presented at the 71st American Academy of Neurology (AAN) Annual Meeting […]
Read More ›Biogen Presents New Data Regarding Adults and Infants with SMA
Biogen has announced it will present new data illustrating the rapidly progressive nature of spinal muscular atrophy (SMA) in adults, adolescents and older children at […]
Read More ›The Winter 2019 issue of Compass in Now Available Online
The Winter 2019 issue of Compass is now available online. This issue covers Cure SMA’s latest drug discovery grant funding and features an updated SMA […]
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