Front Page News
Cure SMA Announces Local Educational Symposiums in 16 US Locations
Cure SMA is pleased to announce a slate of one-day local symposiums, beginning late spring and continuing into fall 2018. The symposiums will be held […]
Read More ›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 […]
Read More ›Advisory Committee on Heritable Disorders in Newborns and Children Recommends Nationwide Newborn Screening for Spinal Muscular Atrophy
The Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) today recommended that newborn screening for spinal muscular atrophy be implemented nationwide. This decision […]
Read More ›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 […]
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