On Saturday, PTC Therapeutics, Inc. announced the presentation of updated interim clinical data from Part 1 of the FIREFISH study investigating risdiplam (RG7916) in babies with Type 1 Spinal Muscular Atrophy (SMA), at the 22nd Annual SMA Researcher Meeting. The data presented by Dr. Baranello demonstrated that at Day 182, over 90% of the babies […]
Today, Cytokinetics announced data from the Phase 2 clinical study of reldesemtiv in patients with SMA, presented in an oral presentation by John W. Day, M.D., Ph.D., Professor of Neurology and Pediatrics (Genetics), Stanford University, at the 2018 Annual Cure SMA Conference in Dallas. This hypothesis-generating study met its primary objective to determine potential pharmacodynamic
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
Biogen to Present New Data at the 2018 Annual SMA Conference Read More »
The 2018 Annual SMA Conference, now in its 30th year, is set to kick off on June 14 and continue into a weekend full of programs and events that bring families, researchers and healthcare professionals together. This year’s conference will be the largest in the Midwest to date. We’re expecting 1,600 attendees, including over 400
The 2018 Annual SMA Conference is Here! Read More »
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
Thank You to Our 2018 Annual SMA Conference Exhibitors! Read More »
As the summer season nears, many families will make plans to travel across the country to exciting destinations or to spend time with those they love. This is no different for those affected by SMA. Although travel can present challenges, regardless of distance or method chosen, below are a variety of resources aimed to guide
Traveling with SMA Read More »
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
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
Ohio Moves One Step Closer to SMA Newborn Screening Read More »
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
Roche Releases Community Statement on Olesoxime Program Read More »
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
