Front Page News
Utah Becomes First State to Implement Permanent Screening for SMA
The Utah Department of Public Health has announced that, effective immediately, all newborns born in the state will be screened for spinal muscular atrophy. This […]
Read More ›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 […]
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 […]
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 […]
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 […]
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 […]
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 […]
Read More ›Scholar Rock Announces Plan to Bring Possible SMA Treatment into Clinical Trials
Scholar Rock, a biotechnology company based in Boston, has announced that they will be investing nearly $50 million to bring SRK-015, a muscle drug for […]
Read More ›Cure SMA Launches Advocacy Action Network
Working together, we’ve achieved tremendous success in advocating for ourselves, our families, and our whole community, particularly in the past few years. Today, we’re building […]
Read More ›Minnesota Becomes Second State to Adopt Newborn Screening for SMA
On December 27, the Minnesota Commissioner of Health accepted the recommendation of the state’s Advisory Committee on Heritable and Congenital Disorders to add SMA to […]
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