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 […]
NIH Releases Summary of the SO-SMART Workshop Read More »
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
Fall 2014 Issue of Directions Now Online Read More »
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
Cytokinetics and Astellas Announce New Phase 2 Clinical Trial Read More »
Just over a month ago, we announced a year-end goal of $540,000 to help fund SMA research and family support. Thanks to the generosity of our community, we’re well on our way to that goal. Less than $20,000 separates us from that benchmark. We think we can do even more next year. Developing a treatment
Help Us Make History with $750,000 for SMA Research Read More »
We’ve just launched a survey to help determine the value and success of our family support and patient services programs. This survey is open to individuals affected by SMA, as well as parents and grandparents of those affected by SMA. Over the next several months, we’ll be evaluating which programs should be kept, and which
Cure SMA Launches Survey on Family Support Programs Read More »
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
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
Results Published on First Large Animal Model of SMA Read More »
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
Registration Opens for the 2015 Annual SMA Conference Read More »
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
Applications Open for 2015 Annual SMA Conference Scholarships Read More »
In February 1994, Barbara and Gene Trainor lost their five-month-old daughter Erin to SMA type I. Throughout the past 20 years, the Trainor family, friends, and colleagues have raised funds for Cure SMA through annual events such as the Chesapeake Crab Feast and the Chesapeake Charity Golf Classic. In addition, they have provided comfort to
Erin Trainor Memorial Fund Reaches Milestone Read More »
