Biogen to Present New Data at the 2018 Annual SMA Conference

Yesterday, Biogen announced it will present data from its SPINRAZA clinical development program –the largest of its kind, with more than six years of data–for spinal muscular atrophy (SMA) at the Cure SMA 2018 Annual SMA Conference in Dallas, TX. “We are proud to support Cure SMA in advancing scientific understanding of the disease to […]

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Thank You to Our 2018 Annual SMA Conference Exhibitors!

Thank you to the following exhibitors who are generously supporting the 2018 Annual SMA Conference. This incredible and unique event would not be possible without your support!  For more information on any of the below companies, please visit our conference exhibitor page.  Conference Exhibitors Platinum Exhibitors Gold exhibitors Convaid Mobility Works Quantum Adaptive Imports Neotech

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Scholar Rock Announces Completion of Dosing of First Cohort in Phase 1 Clinical Trial of SRK-015

Scholar Rock, a biotechnology company based in Boston, recently announced that it has completed dosing of the first cohort and is continuing enrollment of healthy volunteers in its Phase 1 clinical trial of SRK-015. SRK-015, a selective inhibitor of the activation of myostatin, is the company’s lead antibody drug candidate. Scholar Rock believes it has

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Ohio Moves One Step Closer to SMA Newborn Screening

The Ohio Newborn Screening Advisory Committee recommended that SMA be added to their state’s newborn screening panel. The recommendation now goes to the Ohio Department of Health for a rulemaking process. Once these rules are developed and approved, SMA will be officially added to the state panel. During this rulemaking process, SMA advocates will be

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Roche Releases Community Statement on Olesoxime Program

Roche today provided the following community statement on olesoxime. Dear members of the SMA Community, We would like to provide you with an update about olesoxime, an investigational molecule in SMA. Since we bought olesoxime from Trophos in 2015, we have had many difficulties in developing this molecule for people with SMA. These difficulties have

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Full Schedule of Workshops, Panels and Speakers for the 2018 Annual SMA Conference Announced

Cure SMA has released the full schedule of workshops, panels and speakers for the 2018 Annual SMA Conference, to be held at the Hilton Anatole Hotel in Dallas, TX from June 14-17. A detailed timeline with locations are included in the updated schedule, as well as several new workshops like Oral-Motor Exercises for Speech Clarity

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Cure SMA Awards $150,000 Grant to Kathryn Swoboda, MD, Massachusetts General Hospital

Cure SMA has awarded a $150,000 research grant to Kathryn Swoboda, MD, at the Massachusetts General Hospital, for her project, “Clinical and pathologic correlations in patients with early infantile-onset SMA.” SMA is caused by mutations in the SMN1 gene resulting in low levels of SMN protein, which is expressed in every cell in the body.

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2018 Annual SMA Conference Booklet Released

The 2018 Annual SMA Conference Booklet is now posted. The 112-page booklet includes information on workshops, special events and details of the 30th Anniversary SMA Conference, including: 1. A complete schedule, full agenda, and descriptions and speaker bios for the 56 workshops. 2. Details about many of the special events planned during the Annual SMA

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