December 2014

Cure SMA Announces New $50,000 Care Research Grant to Rebecca Davis in the Lab of Dr. Swoboda at University of Utah

Cure SMA has awarded a $50,000 clinical care grant to Rebecca Hurst Davis in the lab of Dr. Kathy Swoboda, for her project focusing on blood sugar levels in SMA. Children with SMA sometimes develop insulin resistance or glucose intolerance, meaning they cannot properly regulate their blood sugar. This project will measure how the blood […]

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NIH Releases Summary of the SO-SMART Workshop

During the Annual SMA Conference in Washington DC this past June, the National Institutes of Health (NIH) hosted a workshop on clinical trial design in SMA for drug developers, clinical trialists, and FDA representatives. The National Institute of Neurological Disorders and Stroke (NINDS) used the meeting as a forum to discuss the appropriate outcome measures

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Fall 2014 Issue of Directions Now Online

The Fall 2014 issue of Directions is now available. 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 120-page issue includes: Information on the upcoming

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Cytokinetics and Astellas Announce New Phase 2 Clinical Trial

Cytokinetics and Astellas jointly announced plans to begin a Phase 2 clinical trial in 2015 in patients with spinal muscular atrophy. The trial will test CK-2127107, a muscle activator. CK-2127107 will be the seventh SMA drug program to advance to clinical trials. Cure SMA provided funding to Cytokinetics for research focused on the potential application

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Cure SMA Announces New $50,000 Clinical Care Research Grant to Nilesh Mehta at Boston Children’s Hospital

We’re excited to announce that we have awarded $50,000 clinical care grant to Dr. Nilesh Mehta, for his project focusing on nutrition in SMA. Some individuals with SMA are at risk for undernutrition, which means they are not receiving enough nourishment. Others are at risk for overnutrition, which means they are consuming too much. Many

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Results Published on First Large Animal Model of SMA

Dr. Arthur Burghes, a Cure SMA Scientific Advisory Board Member from Ohio State University, has published a paper on the first-ever large animal model of SMA. There are a number of unresolved research questions in spinal muscular atrophy, including whether we can correct SMN protein levels when symptoms are present, what is expected of biomarker

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Registration Opens for the 2015 Annual SMA Conference

Registration is now open for the 2015 Annual SMA Conference. The 2015 Annual SMA Conference will be held in Kansas City, MO, at the Westin Crown Center from Thursday, June 18, through Sunday, June 21, 2015. Every year, Cure SMA sponsors a conference to bring together the leading SMA researchers, clinicians, and families living with

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Applications Open for 2015 Annual SMA Conference Scholarships

Applications are now open for scholarships to the 2015 Annual SMA Conference, held June 18-21 in Kansas City, MO. You must complete an application form in order to be considered.  These scholarships are funded through generous donations, fundraisers, and sponsorships. An application deadline will be announced shortly. Decisions will be announced after all applications have

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