Research
Cure SMA Awards $75,000 Grant to Robert Kalb, MD, Northwestern University
Cure SMA has awarded a $75,000 research grant to Robert Kalb, MD, at Northwestern University Feinberg School of Medicine, for his project, “Effects of diminished […]
Cure SMA Awards $150,000 Grant to Umrao Monani, PhD, Columbia University
Cure SMA has awarded a $150,000 research grant to Umrao Monani, PhD, at Columbia University, for his project, “SMA modulators as a means to revealing […]
Biogen Announces New Interim Phase 2 Results From NURTURE
Biogen recently announced new interim Phase 2 results from NURTURE, the ongoing open-label, single-arm study evaluating the efficacy and safety of SPINRAZA or nusinersen among […]
Cure SMA Launches Second Annual Community Survey to Address Important Issues in SMA Treatment
Dear Members of the SMA Community, For the past several years, we’ve been working to collect data and information on our community’s experiences, goals, hopes, […]
Cure SMA Awards $150,000 Grant to Allison Ebert, PhD, Medical College of Wisconsin
Cure SMA has awarded a $150,000 research grant to Allison Ebert, PhD, at the Medical College of Wisconsin, for her project, “Role of astrocyte-produced miR-146a […]
Phase 2 Clinical Trial Results for CK-2127107 Expected in Second Quarter of 2018
Cytokinetics is currently conducting a Phase 2 clinical trial of CK-2127107 – now referred to by the generic name, reldesemtiv – which is designed to […]
Biogen Releases Statement on CHERISH Data and Plans for New Clinical Research
Biogen has provided the following community statement on the final results from CHERISH, a Phase 3 study of SPINRAZA. Dear Members of the SMA community, […]
Winter 2018 Compass Now Available Online
The winter 2018 issue of Compass is now available online. This issue covers Cure SMA’s community survey, and reviews how data is used to improve […]
Preliminary Data from FIREFISH Trial in Type 1 SMA Infants Presented at the International Scientific Congress on Spinal Muscular Atrophy
PTC Therapeutics, Inc. recently announced the presentation of early interim data from Part 1, the dose-finding portion of the FIREFISH study. FIREFISH is a two-part […]
AveXis Releases Community Statement on Expanded Clinical Trials
AveXis has provided the following community statement on AVXS-101. Dear SMA Community, At AveXis, the gene replacement therapy company developing a new approach to treat […]