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 feedback to Cure SMA in relation to several topics. These topics include support programs, patient care, engagement, independence, advocacy, accessibility, education, transitioning to adulthood, and much more. Members of the […]
Cure SMA Establishes an Adult Advisory Council Read More »
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 to all members of the SMA community, including patients, family, friends and caregivers of any age. Ionis will select three entries to use in their annual holiday card, as well
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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 & 5K on September 29, 2019. Team Goin’ For Koen raised a recording breaking $9,000 for the race this year. “We run to represent Cure SMA because they have been there
Team Cure SMA Runners Brave the Rain at the Chicago Half Marathon & 5K Read More »
The Clements Twins, Ava & Leah, are raising money and awareness for spinal muscular atrophy (SMA) through a partnership with Nail & Bone, a socially conscious nail polish company. Their cousin Shane was diagnosed with SMA Type II at 10 months old. “Shane was a very strong and active baby,” according to Regina Philipps, Shane’s
Cure SMA Partners with the Clements Twins and Nail & Bone Read More »
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 older patients (≥ 2 years and < 5 years) with spinal muscular atrophy (SMA) Type 2 achieved a mean increase of 5.9 points from baseline in HFMSE scores, nearly double
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 Society Congress. The 24th Annual International Annual Congress of the World Muscle Society is being held in Copenhagen, Denmark this week. SRK-015 is a highly selective inhibitor of the activation
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 muscular atrophy (SMA) in infants treated pre-symptomatically. Data from the NURTURE study demonstrated that infants who initiated treatment with SPINRAZA prior to the onset of clinical symptoms attained unparalleled results
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 was presented at the 24th International Annual Congress of the World Muscle Society. Presentations include data from the FIREFISH, SUNFISH, and JEWELFISH clinical trials. The SMA program is a collaboration
Paul Guarino was ahead of his time when he started PG Sports as a Twitter page in 2011. Initially, the West Haven, CT native created a simple logo and shirts to promote the newly formed brand. As Guarino gained followers and interest grew in the shirts, he knew it was time for an upgrade. After
Community Spotlight: Paul Guarino of PG Sports Read More »
AveXis, a Novartis company, recently announced that new interim data from the Phase 3 SPR1NT trial in pre-symptomatic patients as well as interim data from the ongoing Phase 3 STR1VE clinical program for Zolgensma showed positive outcomes, demonstrating age‑appropriate major milestone gain with pre‑symptomatic treatment and prolonged event-free survival* in patients with SMA Type 1.
