Cure SMA is expanding its role in spinal muscular atrophy medical affairs at the national level through its Medical Advisory Council (MAC). The MAC sets the agenda for proactive, creative, and collaborative leadership on issues that improve the quality of medical care for those affected by SMA. The focus of the Council is to build
Applications for Cure SMA Medical Advisory Council Due October 15th Read More »
Genentech/Roche recently provided the following community statement with clinical trial updates for SUNFISH (Type 2/3). The SUNFISH trial is advancing to part two, which is designed to be a pivotal, marketing registration trial. Dear SMA Community, We are focused on progressing our studies with our investigational molecule RG7916 with utmost speed and quality and are
Genentech/Roche Releases Clinical Trial Update for RG7916 Read More »
The SMA community celebrated an impactful and advocacy-driven Awareness Month through a number of ways this August. Whether hosting candlelight vigils, meeting with state and local legislatures, holding fundraisers, coordinating MLB awareness games, and more, the community worked together to raise the general publics awareness of SMA. Advocacy Visits On August 2nd, members of the
2017 SMA Awareness Month Recap Read More »
On Tuesday, September 19, at 12:00pm CST (10:00am PST/11:00am MST/1:00pm EST), Cure SMA will hold a webinar updating the community on the status of Spinraza access. Among the topics covered will be: The current status of dosing in states across the US Administration sites, including site list information Commercial and government insurance policies Community resources
Cure SMA to Host Webinar Updating the Community on Spinraza Access 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 470 attendees. The goal of the meeting is to create open communication of early, unpublished data, accelerating the pace of research. The meeting also furthers research by building collaborations—including cross-disciplinary dialogue,
2017 SMA Researcher Meeting Summary: Clinical Research Studies for SMA 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 470 attendees. The goal of the meeting is to create open communication of early, unpublished data, accelerating the pace of research. The meeting also furthers research by building collaborations—including cross-disciplinary dialogue,
During the 2017 SMA Researcher Meeting, we honored one of our 2017 conference champions, Dr. Victor Dubowitz. Dr. Dubowitz’s early contributions to the SMA field were seminal and have been critical to our understanding of SMA pathology and clinical presentation. Dr. Dubowitz, originally from South Africa, is a neurologist, professor emeritus at the Imperial College,
Life & Atrophy, a film about SMA, premiered at the Rhode Island International Film Festival on Saturday, August 12th. It was directed, produced, edited and photographed by Gareth Burghes. The film follows the McIntosh’s, whose son Miles has SMA Type II, as they navigate the diagnosis of SMA and as Miles continues to partake in
SMA Documentary Premieres at 2017 Rhode Island International Film Festival Read More »
Two Cure SMA-funded projects have recently been published. Diane Murrell and her team published the paper, “The Experience of Families With Children With Spinal Muscular Atrophy Type I Across Health Care Systems” in the Journal of Child Neurology. Deborah Boroughs’ paper, “An Evaluation of a Continuing Education Program for Families of Ventilator-Dependent Children with Spinal
Cure SMA-Funded Researchers, Diane Murrell and Deborah Boroughs, Publish Papers 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 470 attendees. The goal of the meeting is to create open communication of early, unpublished data, accelerating the pace of research. The meeting also furthers research by building collaborations—including cross-disciplinary dialogue,
2017 Researcher Meeting Summary: Regulation of Splicing and SMN Protein Expression Read More »
