Author name: Morgan Tuttle

Cure SMA to Host Webinar Updating the Community on Spinraza Access

Leave a Comment / Community Awareness, Front Page News /

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 »

2017 SMA Researcher Meeting Summary: Clinical Research Studies for SMA

Leave a Comment / Conference, Front Page News, Research /

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 The goal of the meeting is to create open communication of early, unpublished data, accelerating the pace of research.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 »

2017 Researcher Meeting Summary: Identification of Candidate Therapeutic Targets and Disease Modifiers

Leave a Comment / Conference, Front Page News, Research /

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 The goal of the meeting is to create open communication of early, unpublished data, accelerating the pace of research.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: Identification of Candidate Therapeutic Targets and Disease Modifiers Read More »

Victor Dubowitz Honored at 2017 Researcher Meeting for Early Contributions to the SMA Field

Leave a Comment / Conference, Front Page News, Research /

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,

Victor Dubowitz Honored at 2017 Researcher Meeting for Early Contributions to the SMA Field Read More »

SMA Documentary Premieres at 2017 Rhode Island International Film Festival

Leave a Comment / Community Awareness, Front Page News /

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 »

Cure SMA-Funded Researchers, Diane Murrell and Deborah Boroughs, Publish Papers

Leave a Comment / Front Page News, Research /

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 »

2017 Researcher Meeting Summary: Regulation of Splicing and SMN Protein Expression

Leave a Comment / Conference, Front Page News, Research /

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 »

Centers for Medicare and Medicaid Services (CMS) Survey

Leave a Comment / Front Page News, Our Impact /

On August 3rd, as part of multiple days of advocacy visits, Cure SMA staff met with leaders from the Medicare division of the Centers for Medicare and Medicaid Services (CMS). The goal of the meeting was to advocate for robust reimbursement for Spinraza, as part of our ongoing coverage and payment policy project. While the

Centers for Medicare and Medicaid Services (CMS) Survey Read More »

Roche Opens US Recruitment for Phase 2 Clinical Trial

Leave a Comment / Clinical Trials, Front Page News /

Roche has opened enrollment in the US on one of their two Phase 2 clinical trials testing RO7034067 (also known as RG7916). Late last year, Roche first announced plans to begin these Phase 2 trials in response to favorable results from a Phase 1 study in healthy volunteers. RO7034067 is an orally available drug that

Roche Opens US Recruitment for Phase 2 Clinical Trial Read More »

SMA Families and Cure SMA Staff Visit Capitol Hill for Advocacy Efforts

Leave a Comment / Advocacy, Front Page News, Our Impact /

On August 2nd, members of the spinal muscular atrophy community kicked off SMA Awareness Month by meeting with a dozen Senate and House congressional offices to advocate for issues that impact our community. The teams conducting these visits included families affected by SMA, and Cure SMA staff. These Senators and Representatives serve on committees important

SMA Families and Cure SMA Staff Visit Capitol Hill for Advocacy Efforts Read More »

Scroll to Top