Research
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 ›Community Update Survey Q&A
Cure SMA recently launched the third annual Community Update Survey to address important issues in SMA treatment and care. The Community Survey covers several areas […]
Read More ›Cure SMA Awards $150,000 Grant to Jocelyn Cote, PhD, University of Ottawa
Cure SMA and Cure SMA Canada have awarded a $150,000 research grant to Jocelyn Cote, PhD, at the University of Ottawa for his project, “Investigating […]
Read More ›Cure SMA Awards $200,000 Grant to Alberto Kornblihtt, PhD, Universidad de Buenos Aires
Cure SMA and FAME (Families of SMA, Argentina) have awarded a $200,000 research grant to Alberto Kornblihtt, PhD, at the Universidad de Buenos Aires, Argentina, […]
Read More ›Cure SMA Awards $200,000 Grant to Charlotte Sumner, MD, Johns Hopkins University
Cure SMA has awarded a $200,000 research grant to Charlotte Sumner, MD, at Johns Hopkins University, for her project, “Neurofilaments as markers of neurodegeneration in […]
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