Search Results: zolgensma
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 […]
Read More ›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) […]
Read More ›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 […]
Read More ›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 […]
Read More ›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 […]
Read More ›Updated SMA Drug Pipeline Now Available
We’ve recently released an update to the SMA drug pipeline. This latest version includes: 23 active programs, including two approved therapies. 15 pharmaceutical partners. 6 […]
Read More ›Family Friendly Research Poster Session Presenters Announced
The Family Friendly Research Poster Session will be held on Saturday, June 29th from 6:30pm to 8:00pm at the 2019 Annual SMA Conference. The Family […]
Read More ›Cure SMA and Parent Project Muscular Dystrophy Announce Collaboration
Today, Cure SMA and Parent Project Muscular Dystrophy (PPMD) announced their strategic collaboration to collect real-world data to improve the lives of those affected by […]
Read More ›Cure SMA Advocates for SMA Community at ICER Hearing
On Thursday, March 7, Cure SMA testified on behalf of the SMA community at a meeting of the Institute for Clinical and Economic Review (ICER). […]
Read More ›Fall 2018 Issue of Directions Now Available
The Fall 2018 issue of Directions is now available online. Please visit our Support & Care Publications page to download this issue or past issues […]
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