The 8th Annual “Hope on the Hill” Congressional Dinner was held on Tuesday, November 27, 2018 at The Willard Hotel in Washington, DC. More than 170 people from the SMA community – including government and industry partners – attended the dinner which raised $200,000 to fund advocacy efforts, research, and family support services. Since 2011, […]
Hope on the Hill Congressional Dinner Raises $200,000 Read More »
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 presentation is also available for download. An update on the status of dosing for Spinraza across the United States included the following information: As of October 2018, ~2,700 patients have
December Webinar on SMA Treatments and Clinical Trials Now Available Online Read More »
Over our past fiscal year—from July 1, 2017, to June 30, 2018—Cure SMA has funded over $5 million in new research funding. This funding will be used strategically to help accelerate research, and ensure we are developing treatments for all types, ages, and stages of SMA. The areas funded include: Basic research, with a particular focus
2018 Research Year-in-Review Read More »
An important goal of our research funding strategy is to share scientific findings with the broader scientific community. Scientists who receive Cure SMA funding often publish their findings in peer-reviewed journals. This means that other scientists can learn from their results, which will pay dividends across the wider landscape of SMA research—allowing us to multiply
Cure SMA Funding Leads to 16 New Research Publications in 2018 Read More »
When Milo Berkovits was seven months old, his daycare teacher noticed he was not reaching his milestones. What followed from there on were visits to three different pediatricians, an early intervention assessment, a review by a private occupational therapist, another assessment by a private physical therapist and finally a neurologist visit. At nine months old,
Community Spotlight: The Berkovits Family Read More »
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 moment to look back at all our successes. Thank you to all our families and advocates for your support and continued efforts to cure SMA. We wish you all a
Advocacy Year-in-Review 2018 Read More »
Bigoen has provided the below community statement on Spinraza. Dear Members of the SMA community, As we approach the two-year anniversary of the U.S. Food and Drug Administration’s (FDA) approval of SPINRAZA® (nusinersen), we want to thank you for your ongoing support. We share your commitment to advancing research to improve the lives of those
Biogen Issues Q4 Community Statement on Spinraza Read More »
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 Community, We are providing this update to the SMA community in the U.S. at the request of Cure SMA. Following our recent October update about our Biologics License Application (BLA)
AveXis Issues Community Statement on BLA Acceptance Read More »
The SMA community has multiple victories to celebrate as the year comes to an end. Thank you to all our amazing families and advocates who have and continue to make this progress possible. Virginia Adopts SMA Newborn Screening Last month, Virginia’s Newborn Screening Advisory Committee adopted SMA to their Newborn Screening Panel. The state government
The fall 2018 issue of Compass is now available online. This issue of Compass covers the SMA Care Center Network and its goal of developing an evidence-based standard of care to improve the lives of people with SMA. SMA Care Center Network In October, Cure SMA launched the SMA Care Center Network, a collection of specialized
Fall 2018 Issue of Compass Now Available Read More »
