Research
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 […]
SMA Industry Collaboration Releases Spinal Muscular Atrophy Voice of the Patient (VoP) Report
Cure SMA and our partners in the SMA Industry Collaboration are pleased to announce the release of the Spinal Muscular Atrophy Voice of the Patient […]
2018 Updated SMA Drug Pipeline Released
We’ve recently released an update to the SMA drug pipeline. This latest version includes: 16 active programs, including one approved therapy. 14 pharmaceutical partners. 6 programs […]
AveXis Announces Expanded Clinical Development Program for AVXS-101 in Spinal Muscular Atrophy
AveXis, a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today provided an overview of the expanded […]