Cure SMA Releases Status Update on Data Accuracy Issues with Zolgensma

Leave a Comment / Front Page News /

Yesterday, the FDA issued a statement identifying data accuracy issues with Avexis’ SMA therapy, Zolgensma. Cure SMA is in communication with the FDA and is committed to representing the SMA patient voice and ensuring it is represented in any and all discussions. While the manipulation of data is a very serious matter, the agency has re-reviewed the […]

Cure SMA Releases Status Update on Data Accuracy Issues with Zolgensma Read More »

FDA Issues Statement on Data Accuracy Issues with Recently Approved Gene Therapy

Leave a Comment / Front Page News /

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 further details. We will be issuing updates to the community as we have news to share. The FDA’s statement reads: As a public health agency, we believe that it is

FDA Issues Statement on Data Accuracy Issues with Recently Approved Gene Therapy Read More »

Biogen Issues Community Statement on Spinraza

Leave a Comment / Clinical Trials, Front Page News /

Biogen has provided the following community statement on Spinraza. Dear members of the SMA community, Following a busy spring of data presentations at medical congresses and the annual Cure SMA Conference, we are pleased to provide updates on research highlighting SPINRAZA® (nusinersen) data and real-world experience for individuals with spinal muscular atrophy (SMA). Recent updates

Biogen Issues Community Statement on Spinraza Read More »

Biogen Announces New NURTURE Data at the 2019 Annual SMA Conference

Leave a Comment / Clinical Trials, Front Page News, Research /

Biogen today announced new results from the NURTURE study, adding data to the longest study of spinal muscular atrophy (SMA) in pre-symptomatic infants (n=25). These data reported, after up to 45.1 months of analysis, continue to demonstrate efficacy and safety in patients treated pre-symptomatically with SPINRAZA in comparison to the natural history of this disease.

Biogen Announces New NURTURE Data at the 2019 Annual SMA Conference Read More »

Updated SMA Drug Pipeline Now Available

Leave a Comment / Front Page News, Our Impact, Research /

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 programs in clinical trials. An ever-increasing breadth of potential treatment approaches to SMA. Effective Treatments for All Ages, Stages and Types of SMA The updated version of this pipeline reflects

Updated SMA Drug Pipeline Now Available Read More »

Cytokinetics Releases New Data from Reldesemtiv Clinical Study at the 2019 Annual SMA Conference

Leave a Comment / Clinical Trials, Front Page News, Research /

Cytokinetics, Incorporated, today announced that data from two preclinical studies of reldesemtiv were presented at the 2019 Annual Cure SMA Conference in Anaheim, CA, showing that the addition of reldesemtiv to treatment with SMN upregulators (nusinersen and SMN-C1, an analogue to risdiplam) significantly increased muscle force in a mouse model of spinal muscular atrophy (SMA).

Cytokinetics Releases New Data from Reldesemtiv Clinical Study at the 2019 Annual SMA Conference Read More »

Cure SMA Receives $150,000 Grant From the Oscar G and Elsa S Mayer Family Foundation

Leave a Comment / Front Page News, Support & Care /

Cure SMA is pleased to announce receipt of $150,000 grant over three years, awarded by the Oscar G. and Elsa S. Mayer Family Foundation to support continued development of the SMA Care Center Network. The mission of the SMA Care Center Network is to: Improve SMA clinical care and disease management, leading to creation of

Cure SMA Receives $150,000 Grant From the Oscar G and Elsa S Mayer Family Foundation Read More »

Scroll to Top