Latest News
Federal Committee to Vote on Spinal Muscular Atrophy Newborn Screening on Thursday
On Thursday, February 8, the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC), a federal committee that oversees newborn screening, will vote on whether to recommend newborn screening […]
Read More ›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 seamless, open-label, multi-center study to […]
Read More ›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 known as AVXS-101, we […]
Read More ›Cure SMA to Host Webinar on Treatment Access and Clinical Trials on February 15
On Thursday, February 15, at 12:00pm CST (10:00am PST/11:00am MST/1:00pm EST), Cure SMA will hold a webinar updating the community on treatment access and clinical trials. Among the topics covered […]
Read More ›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 (VoP) Report. This report is […]
Read More ›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 in clinical trials. An ever-increasing […]
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