August 2017

2017 SMA Researcher Meeting Summary: Clinical Research Studies for SMA

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 The goal of the meeting is to create open communication of early, unpublished data, accelerating the pace of research.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

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

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 The goal of the meeting is to create open communication of early, unpublished data, accelerating the pace of research.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

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

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,

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SMA Documentary Premieres at 2017 Rhode Island International Film Festival

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

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

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

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

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,

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Centers for Medicare and Medicaid Services (CMS) Survey

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

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Roche Opens US Recruitment for Phase 2 Clinical Trial

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

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

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

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2017 SMA Researcher Meeting Summary: SMA Therapy Development

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,

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