The SMART study supplements a growing body of evidence on the use of Zolgensma in a patient population older and heavier (1.5 – 9.1 years of age) than the children treated in previous clinical studies1-6 Nearly all patients treated maintained or improved motor milestones after 52 weeks, with most switching to the one-time gene […]
Today is Rare Disease Day and the rarest day of all, as this year’s occurrence takes place on February 29! In the U.S., spinal muscular atrophy (SMA) affects nearly 9,000 people. Yet, the SMA community is part of a greater community, the rare disease community, that comprises of 300 million people worldwide. Together, we create
Celebrate Rare Disease Day with Cure SMA Read More »
Today, on Rare Disease Day, U.S. Transportation Secretary Pete Buttigieg and White House officials announced actions the U.S. Department of Transportation (DOT) plans to take to improve air safety for individuals who use wheelchairs, including passengers with spinal muscular atrophy (SMA). Cure SMA attended the February 29th White House announcement and plans to submit public
Cure SMA Applauds DOT Proposal to Make Air Travel Safer for Wheelchair Users Read More »
Each year, Cure SMA invites scientists from around the world to submit funding proposals for basic research projects that address specific unanswered questions in SMA biology. Our Scientific Advisory Board then ranks the submitted proposals on both their scientific merit and relevance to Cure SMA’s research priorities. Funding is awarded to the highest ranked projects.
Each year, Cure SMA invites scientists from around the world to submit funding proposals for basic research projects that address specific unanswered questions in SMA biology. Our Scientific Advisory Board then ranks the submitted proposals on both their scientific merit and relevance to Cure SMA’s research priorities. Funding is awarded to the highest ranked projects.
Cure SMA Awards $150,000 Grant to Melissa Bowerman, PhD, at Keele University Read More »
Cure SMA released a national report on National Caregivers Day that chronicles the caregiving challenges experienced by individuals with spinal muscular atrophy (SMA) and recommends state and federal actions to help address their caregiver needs. Stuck Inside: A National Report on Caregiving for Individuals with SMA features numerous first-person experiences and quotes highlighting the essential
New Report Highlights Caregiving Challenges of Individuals with Spinal Muscular Atrophy Read More »
Led by Keishla Rolon, Ramon Rivera Vega, Elizabeth Serrano, Natasha Santiago, and Brenda Luciano, Cure SMA’s Puerto Rico chapter has already been hard at work helping individuals and families throughout the island. In late January, the Puerto Rico community came together to celebrate their new chapter through Summit of Strength and Walk-n-Roll events, with over
Cure SMA is Thrilled to Announce the Establishment of our New Chapter in Puerto Rico! Read More »
In a world where adversity often brings out the best in people, the Stickane family of Southlake, TX stands as a shining example of unwavering love and resilience. Their journey began when their middle son, Luke, was diagnosed with SMA in 2017. Luke’s diagnosis with SMA was a turning point for the Stickane family
Leland Ianno is eight years old, but he’s an old soul with a deep curiosity, a love of people, and a passion for history. His mom, Holly, says “Leland is fascinated by historical times but also the history of the people around him. He wants to know everything, and he’ll sit and have a conversation
Community Spotlight: Holly Ianno and Family Read More »
You did it! We’ve now reached our goal of 100 percent newborn screening of spinal muscular atrophy (SMA) in all 50 states with the addition of Hawaii at the start of 2024. You made this accomplishment possible by advocating and making the case at the state and national level for universal newborn screening. Your
100% of States Now Screening Newborns for SMA Read More »
