On Wednesday, August 28th, at 12pm CST (10am PST/11 am MST/1pm EST) Cure SMA will host a webinar to provide updates on Zolgensma. The webinar will be divided into three separate sections. We’re pleased to have representatives from the FDA, Cure SMA, and AveXis, a Novartis Company, participating to share information and answer questions. Panelists […]
Cure SMA to Host August 28th Webinar on Zolgensma Updates Read More »
Thank you to everyone who attended the 2019 Annual SMA Conference at the Disneyland in Anaheim, California! It was an impactful weekend of community, research and fun. We are happy to announce that, including on-site registration, there were over 2,500 attendees in total – our largest conference to date! It was such an honor to
Thank You for a Wonderful 2019 Annual SMA Conference! Read More »
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 The goal of the meeting is to create open communication of early, unpublished data, accelerating the pace of research.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 »
On April 18, 2017, the SMA community—families, clinicians, researchers, industry and regulators—gathered for a Patient-Focused Drug Development (PFDD) Meeting with the FDA. As part of the reauthorization of the Prescription Drug User Fee Act (PDUFA), the FDA is required to gather community feedback more systematically, through events such as this PFDD meeting, and incorporate that
