Read the Summer 2015 Issue of Compass

The latest issue of Compass, our research publication, is now available online. This summer 2015 issue includes information on our recent drug discovery grants, plus an update to our SMA drug pipeline. Developing a treatment and cure for SMA is the driving force behind Cure SMA, and the goal of our three-stage research model. Drug […]

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The 2015 Annual SMA Conference is Here!

The 26th Annual SMA Conference kicks off tomorrow with our Newly Diagnosed Program, and continues into the weekend with all of our usual programs that bring families, researchers, and healthcare providers together to network, learn and —plus a few new initiatives. This will be one of our largest conferences ever. We’re expecting over 1,300 attendees

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2015 Annual SMA Conference Exhibitors

Thank you to the following exhibitors who have generously supported our 2015 Annual SMA Conference. This one-of-a-kind event would not be possible without your support! For more information on any of these companies, please visit our conference exhibitor page. Platinum Ki MobilityQuantum Rehab Gold 3E Love Bio-Rad Laboratories, Inc. Convaid Neotech Products Percussionaire Permobil PromptCare

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Cure SMA Releases New Clinical Trial Care Series Booklet

As more and more SMA drug programs progress through clinical trials, there is an increasing need for accurate and unbiased information on clinical trials from our community. Cure SMA has been working to address this through education efforts targeted to the specific needs our community. The centerpiece of these education efforts is our new care

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Isis Releases Encouraging Results from Phase 2 Study

Isis Pharmaceuticals provided an update on its ongoing open-label Phase 2 clinical study of ISIS-SMNRx in infants with Type I spinal muscular atrophy (SMA). Previously the company reported data from this study on event-free survival, measures of muscle function and assessments of developmental milestones. The data reported now shows continued increases in median event-free survival

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SMA Drug Pipeline Grows to 18 Programs

We’ve recently released an update to our SMA drug pipeline. This latest version shows the continued growth of the pipeline, setting several new marks for both breadth and depth: We have 18 active programs. We have 15 pharmaceutical partners. We have seven programs in clinical trials. The cumulative pipeline total is 26 programs, including eight

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Spring 2015 Directions Posted Online

The Spring 2015 issue of Directions, our biannual family support newsletter, builds our community by sharing stories and experiences. Families from all over the world connect with each other through Cure SMA to chat, organize, and share stories, photos, news, information, and—most importantly—hope. This issue includes information on the upcoming 2015 Annual SMA Conference, news

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Biogen Announces EMBRACE, a New Phase 2 Clinical Trial

Biogen, in partnership with Isis Pharmaceuticals, has announced a new Phase 2 clinical trial, called EMBRACE. EMBRACE is a multi-center, Phase 2 clinical study evaluating the safety and exploratory efficacy of the investigational drug, ISIS-SMNRx, (ISIS 396443) in patients with infantile or childhood-onset Spinal Muscular Atrophy (SMA). The study is a double-blind, randomized, sham-procedure controlled

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2015 Annual SMA Conference Booklet Now Posted

The 2015 Annual SMA Conference Booklet has been posted. The booklet includes: A complete schedule, including information about the special events we’ve got planned for each evening. Times, locations, details, and speaker bios for all of our exciting workshops. Information on our sponsors and exhibitors, who have generously supported this conference. And more! Even if

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High School Students Lead the Way at Concert for a Cure

The 15th Annual Concert for a Cure was held on Saturday, May 9, 2015, a the Diablo Country Club in Danville, CA and was a tremendous success raising over $150,000 for a treatment and cure. The evening was made rewarding for donor and families when Dr. John Day, Stanford Professor for Neurology, Pediatrics, and Pathology,

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