Latest News
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 research, care and coverage in […]
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 is an important step toward […]
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 announcement makes Utah the first […]
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 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 […]
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