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 »
Novartis today announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s Biologics License Application (BLA) for AVXS-101, now known as ZOLGENSMA® (onasemnogene abeparvovec-xxxx)1, an investigational gene replacement therapy for the treatment of spinal muscular atrophy (SMA) Type 1. ZOLGENSMA is designed to address the genetic root cause of SMA Type 1,
Novartis Announces FDA Filing Acceptance and Priority Review for AVXS-101 Read More »
On Wednesday, December 12th, at 12:00pm CST (10:00am PST/11:00am MST/1:00pm EST) Cure SMA will hold a webinar updating the community on the latest information on SMA treatments and trials. Topics covered will include: AveXis’s BLA filing with the FDA Status of access to Spinraza Ongoing and upcoming SMA clinical trials As with previous discussions, the
Register for Cure SMA’s Webinar on SMA Treatments and Clinical Trials Read More »
