In 2018, Spinal Muscular Atrophy (SMA) was added to the federal Recommended Uniform Screening Panel (RUSP), a panel of diseases that newborns in the United States are recommended to be screened for. However, the decision on whether to implement newborn screening for a specific condition is up to each state. Since SMA’s inclusion on the […]
Cure SMA Newborn Screening Registry (NBSR) Read More »
A recording of last week’s webinar on Zolgensma updates is now available on Cure SMA’s YouTube channel. A presentation of the webinar can be found here. Panelists from the August 28th webinar included Dr. Peter Marks, M.D. Ph.D., Director, Center for Biologics Evaluation and Research (CBER) at FDA; Kenneth Hobby, President at Cure SMA; Mary
Zolgensma Update and Approval Webinars Now Available Online Read More »
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 The goal of the meeting is to create open communication of early, unpublished data, accelerating the pace of research.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
2019 Researcher Meeting Summary: Clinical Research Studies for SMA Read More »
Twenty Team Cure SMA runners completed the 2019 Falmouth Road Race on Sunday, August 18. Together, the runners successfully raised over $52,000 for Cure SMA, the most in the race’s history for Team Cure SMA. The race celebrated its 47th annual run this year and brought more than 150 non-profit organizations together to raise money
Team Cure SMA Completes the 2019 Falmouth Road Race in Cape Cod Read More »
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 The goal of the meeting is to create open communication of early, unpublished data, accelerating the pace of research.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
2019 SMA Researcher Meeting Summary: Basic Research Podium Presentations Read More »
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 »
