Search Results: Zolgensma
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 […]
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 […]
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 […]
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 […]
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). […]
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 […]
December Webinar on SMA Treatments and Clinical Trials Now Available Online
A recording of the December 13th webinar updating the community on SMA treatments and clinical trials is now available online. A PDF of the webinar […]
Advocacy Year-in-Review 2018
For Cure SMA, 2018 has been a year of great progress. As we continue to make strides into 2019, it is important to take a […]
AveXis Issues Community Statement on BLA Acceptance
AveXis has provided the following community statement on the BLA submission and acceptance for their investigational therapy, AVXS-101 – now known as ZOLGENSMA®. Dear SMA […]
Novartis Announces FDA Filing Acceptance and Priority Review for AVXS-101
Novartis today announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s Biologics License Application (BLA) for AVXS-101, now known as ZOLGENSMA® […]
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