Author name: Sarah McCall

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. 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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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) 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

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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 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

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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 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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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 & 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

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Cure SMA Partners with the Clements Twins and Nail & Bone

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

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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 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

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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 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

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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 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

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