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, […]
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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
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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 »
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 share another story about Steven Verdile of New York. Toward the end of high school, Steven Verdile faced a reality many young adults have experienced: he
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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
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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
