November 2018

Register for Cure SMA’s Webinar on SMA Treatments and Clinical Trials

On Wednesday, December 12th, at 12:00pm CST (10:00am PST/11:00am MST/1:00pm EST) Cure SMA will hold a webinar updating the community on the latest information on SMA treatments and trials. Topics covered will include: AveXis’s BLA filing with the FDA Status of access to Spinraza Ongoing and upcoming SMA clinical trials As with previous discussions, the […]

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Scholar Rock Announces Publication of Preclinical Data on the Therapeutic Benefit of Inhibiting Myostatin Activation in Models of SMA

Scholar Rock, a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced the publication, “Specific Inhibition of Myostatin Activation is Beneficial in Mouse Models of SMA Therapy” in the peer-reviewed journal Human Molecular Genetics. The publication details preclinical studies demonstrating that a highly

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Cure SMA Launches “SMArt Moves” for Early Diagnosis and Treatment

Cure SMA today launches SMArt Moves, a new disease awareness and educational campaign to empower parents, pediatricians and other healthcare professionals to swiftly recognize and diagnose the early signs of motor delays. This allows infants and children diagnosed with SMA (spinal muscular atrophy) to receive life-changing—and in some cases, life-saving—treatment. Infants and toddlers diagnosed with

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8th Annual “Hope on the Hill” Congressional Dinner Will Be Held Later This Month in Washington D.C.

The 8th Annual “Hope on the Hill” Congressional Dinner will be held on Tuesday, November 27, bringing together SMA families, government officials, and industry leaders to look forward to the next stages for our community. Through this event, we hope to build momentum in the Washington D.C. area to increase awareness of our mutual goals

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Cure SMA is Thrilled to Launch a New Support Program for Teens and Adults with SMA

Along with funding SMA research and care, Cure SMA provides thousands of individuals and families with vital family support and resources that help people navigate life with SMA. In addition to our current support programs, Cure SMA is thrilled to launch a brand-new program specifically for teens and adults living with SMA. This program is

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Summit of Strength Program Receives Positive Feedback and Increases Attendance

Attendance and feedback have been extremely positive for this new program. The Cure SMA Summit of Strength Program kicked off in July and has continued to grow with great success! The Summit of Strength is a day of community and learning; it brings together families in their local areas to discuss local SMA care, support

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