Front Page News
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 […]
Read More ›Risdiplam Data Presented at World Muscle Society Congress Demonstrates Continued Benefit for Patients with Spinal Muscular Atrophy
Today, new data was presented demonstrating the ongoing benefit of risdiplam (RG7916) for the treatment of all types of spinal muscular atrophy (SMA). The data […]
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