Genentech, a member of the Roche Group, today announced positive data from the pivotal Part 2 of the SUNFISH study evaluating risdiplam in people aged 2-25 years with Type 2 or 3 spinal muscular atrophy (SMA). The study met its primary endpoint of change from baseline in the Motor Function Measure 32 (MFM-32) scale after […]
This guest post is written by April Leiffer Henry If you ask 4-year-old Winnie Stanley what she wants to be when she grows up, she’ll say a superhero. But to many people in West Virginia, this sweet, adorable little fighter already is a superhero. Winnie is a preschooler whose favorites are Play-Doh, the color pink,
A Tiny Warrior: Winnie Stanley’s Story Read More »
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. We are pleased to close out our recognition of this month with a story from Angela Wrigglesworth of Texas. On the first day of each new school year, Angela Wrigglesworth
Community Spotlight: Angela Wrigglesworth Read More »
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) placed a partial hold on clinical trials for intrathecal (IT) administration of AVXS-101. This partial hold by the FDA only impacts the IT formulation and does not impact the currently
AveXis Issues Community Statement on AVXS-101 Clinical Hold Read More »
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 clinical trials for intrathecal administration of AVXS-101. The announcement follows an AveXis communication to health authorities and clinical trial investigators based on findings from a small, AveXis-initiated pre-clinical study in
Novartis Announces AVXS-101 Intrathecal Study Update Read More »
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
Cure SMA and Ionis Announce Holiday Card Contest Read More »
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 »
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
