Search Results: Zolgensma
Novartis Issues Community Statement with Update on AVXS-101 Intrathecal Clinical Development Program
Dear SMA Community, We recently received important feedback from the U.S. Food and Drug Administration (FDA) following their review of data from the STRONG study […]
Community Spotlight: Braedon’s Newborn Screening Diagnosis
September is Newborn Screening Awareness Month, a chance to talk about the progress we have made in getting spinal muscular atrophy (SMA) added to newborn […]
Thank You for a Wonderful 2020 Virtual SMA Conference!
Cure SMA would like to thank everyone who attended last week’s 2020 Virtual SMA Conference! It was an impactful week of online workshops, networking, and […]
2020 Virtual SMA Conference Booklet is Now Available Online
Cure SMA is pleased to announce that the 2020 Virtual SMA Conference booklet is now available for download. The booklet includes important details on the […]
Community Statement from AveXis Regarding Commitment to SMA Community Amid COVID-19 Pandemic
Dear SMA Community, Like many organizations across the United States and around the world, AveXis is closely monitoring the coronavirus […]
Cure SMA Working Group Revises Recommendations for the Treatment of Infants Diagnosed with SMA via Newborn Screening Who Have 4 Copies of SMN2
To help clinicians and the families they serve in the decision of when to administer therapy to infants identified with SMA via newborn screening, Cure […]
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) […]
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 […]
2019 SMA Researcher Meeting Summary: Clinical Drug Development
The annual SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA. This year, we had a record setting 735 […]
FDA Issues Statement on Data Accuracy Issues with Recently Approved Gene Therapy
Today, the FDA issued the below statement identifying data accuracy issues with AveXis’ SMA therapy, Zolgensma. Cure SMA is investigating this issue and will report when we have […]