On July 3, 2018, Health and Human Services Secretary Alex Azar approved the recommendation that newborn screening for spinal muscular atrophy be implemented nationwide. This recommendation was issued on February 8, 2018, by the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC). With Secretary Azar’s signature, SMA becomes part of the Recommended Uniform Screening Panel […]
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 attendees join together in Anaheim, CA. The goal of the meeting is to create open communication of early, unpublished data, accelerating the pace of research. The meeting also furthers research
Earlier this week, the FDA issued a statement identifying data accuracy issues with AveXis’ SMA therapy, Zolgensma. Cure SMA immediately opened an investigation into the matter and is committed to representing the SMA patient voice and ensuring it is represented in any and all discussions with the FDA, Novartis, and AveXis. Below is an update
Cure SMA Update on Data Accuracy Issues with AveXis’ SMA Therapy, Zolgensma Read More »
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 »
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 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 »
SMA Awareness Month occurs in August! The SMA community officially recognizes August as Spinal Muscular Atrophy Awareness Month and Cure SMA has been coordinating activities since 1996. August is a great time to spotlight the work our community does year-round to raise awareness of and funds for SMA. In addition to our usual work –
SMA Awareness Month 2019 Has Begun! Read More »
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 »
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, 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).
