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Cure SMA

BY: Sammi G | Dec 11, 2018

curesma Spinal muscular atrophy (SMA) is a disease that robs people of strength by affecting the motor nerve cells in the spinal cord, taking away the ability to walk, eat, or breathe. SMA can affect any race or gender. It is the number one genetic cause of death for infants, and approximately 1 in every 50 individuals is a genetic carrier for SMA. But there’s great reason for hope. We know what causes SMA and what we need to do to develop effective therapies, and we’re on the verge of major breakthroughs that will strengthen our children’s bodies, extend life, and eventually lead to a cure. There are now 17 ongoing drug programs for SMA, over half of which have been funded by Cure SMA. This means we’re getting closer and closer to an FDA-approved therapy for people affected by SMA. Earlier this year, recruiting opened for the first-ever Phase III clinical trial of a drug that may treat the underlying cause of SMA. Cure SMA was one of the original funders for this project. In total, we’ve invested $57 million in SMA research. While there’s great promise in the research landscape, there’s also a pressing need for continued and growing investment. We must keep building up a breadth and depth of possible treatment options, so that we don’t lose any of the momentum we’ve created, or waste any time when we see an opportunity that may lead to a cure for SMA. We won’t stop working toward a world without SMA, but until we have a treatment and cure, we’ll do everything we can to improve quality of life for children and families affected by the disease today. Whether through informational packets, care packages, our equipment pool—or any of our services that reach over 3,500 families annually—we provide them the resources they need to live active, engaged, and hopeful lives today. We also host the Annual SMA Conference each year. This conference brings together researchers, healthcare professionals, and families to network, learn, and collaborate. The conference is the largest in the world focused specifically on SMA, and it attracts the top scientists and companies in the field. We have more than 110,000 members and supporters, with 31 volunteer chapters throughout the country.We host approximately 300 fundraising and awareness events annually. Learn about how you can join us in reaching a treatment and cure at

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