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

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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

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 The goal of the meeting is to create open communication of early, unpublished data, accelerating the pace of research.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

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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

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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

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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

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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 SMA Researcher Meeting Summary: Special Session on Fatigue and Muscle Weakness in SMA

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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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Cure SMA Receives Donation from the Bugenske Family

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Cure SMA recently received a donation of 14 special tomato feeder seats and 80 large sheepskin blankets from the Bugenske family. The feeder seats were immediately sent out, and helped Cure SMA fulfill the requests of each family on the equipment waiting list. The donation of large sheepskin blankets helps fund a new support program,

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August is SMA Awareness Month!

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The SMA community officially recognizes August as Spinal Muscular Atrophy Awareness Month, an effort that Cure SMA has been coordinating since 1996. Throughout August, the community works together to raise the general public’s awareness of SMA by hosting candlelight vigils, contacting their local legislature, holding fundraisers, coordinating MLB awareness games, and providing information to local

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