Front Page News
Community Spotlight: Angela Wrigglesworth
October has been National Disability Employment Awareness Month, and throughout the month, you have heard stories about adults in the workforce who live with SMA. […]
Read More ›AveXis Issues Community Statement on AVXS-101 Clinical Hold
Dear SMA community, Today, we announced an AVXS-101 intrathecal study update. In the update, we shared that the United States Food & Drug Administration (FDA) […]
Read More ›Novartis Announces AVXS-101 Intrathecal Study Update
The below statement is a press release from Novartis and AveXis. Novartis today announced the United States Food & Drug Administration (FDA) placed a partial hold on […]
Read More ›Community Spotlight: Steven Verdile
October has been National Disability Employment Awareness Month, and throughout the month, you have heard stories about adults in the workforce who live with SMA. […]
Read More ›Cure SMA Establishes an Adult Advisory Council
We are happy to announce the establishment of the Adult Advisory Council. The Adult Advisory Council is a volunteer council that provides guidance, advice, and […]
Read More ›Cure SMA and Ionis Announce Holiday Card Contest
We are excited to share that Cure SMA and Ionis Pharmaceuticals are once again teaming up for a holiday card contest! This contest is open […]
Read More ›Team Cure SMA Runners Brave the Rain at the Chicago Half Marathon & 5K
Twenty Team Cure SMA runners braved the pouring rain and successfully raised over $12,000 for Cure SMA while running in the Chicago Half Marathon & […]
Read More ›AveXis Presents Updated STRONG Data Demonstrating a Higher Mean Increase in Hammersmith Functional Motor Scale-Expanded (HFMSE) Scores Among Older SMA Type 2 Patients Following One-time Intrathecal Administration of AVXS-101
AveXis, a Novartis company, announced on October 5, 2019 new interim data from the Phase 1/2 STRONG study for intrathecal (IT) administration of AVXS-101, demonstrating […]
Read More ›Scholar Rock to Present Preclinical and Phase 1 Clinical Data on SRK-015 at the World Muscle Society Congress
Scholar Rock recently announced that a poster presentation highlighting preclinical and Phase 1 healthy volunteer data for SRK-015 will be presented at the World Muscle […]
Read More ›Landmark NURTURE Study of Infants with Spinal Muscular Atrophy (SMA) Treated Pre-Symptomatically with Spinraza Published in Neuromuscular Disorders
Biogen today announced that the journal Neuromuscular Disorders has published data from NURTURE, the first study investigating a treatment targeting the underlying cause of spinal […]
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