While sibling relationships aren’t always easy, Alexandra ‘Lexi’ Lakhman and her brother Joe have always been close. Despite Lexi being six years older than Joe, she grew up viewing Joe as a role model, particularly for his positive outlook on life. Joe was diagnosed with spinal muscular atrophy (SMA) Type 2 in 2002 when there […]
Community Spotlight: Alexandra and Joe Lakhman Read More »
As treatment options increase for adolescents and young adults living with SMA, we need tools to measure the effectiveness of these treatments from the patient’s perspective. Dr. Chad Heatwole and his team at the University of Rochester have developed the Spinal Muscular Atrophy Health Index (SMA-HI) for this purpose.1 The SMA-HI is an SMA-specific survey
The recent availability of new treatments for SMA would not have been possible without the participation of SMA-affected individuals in clinical trials. Yet participating in clinical trials can be stressful. We at Cure SMA want to understand how future trials can be designed to reduce stress for participants and their parents and caregivers. To achieve
In June, SMA researchers and clinicians from around the world met in Anaheim, CA, for the 2022 Annual SMA Research and Clinical Care Meeting. There they shared their most recent data with the goal of accelerating the pace of research into SMA treatments and achieving excellence in SMA clinical care. Below is a summary of
2022 Cure SMA Annual Research Meeting Summaries – Special and Clinical Research Session Read More »
In June, SMA researchers and clinicians from around the world met in Anaheim, CA, for the 2022 Annual SMA Research and Clinical Care Meeting. There they shared their most recent data with the goal of accelerating the pace of research into SMA treatments and achieving excellence in SMA clinical care. Below is a summary of
2022 Cure SMA Annual Research Meeting Summaries – Basic Research Sessions Read More »
Congress has recognized the unmet and new needs of individuals with SMA by including legislative language in must-pass legislation asking the National Institutes of Health (NIH) to continue research in SMA. At the 2022 Cure SMA conference and throughout the summer, individuals with SMA and their families educated Congress about existing challenges faced by the
Cure SMA Advocacy Campaign Leads to Congressional Support for More SMA Research Read More »
On Thursday, August 4, 2022, six members of the SMA community will have the opportunity to talk directly with the U.S. Food and Drug Administration (FDA) about their lived experiences with SMA and their unmet medical needs. This Patient-Led Listening Session will be a small, private teleconference attended by patient advocates, FDA representatives from the
Cure SMA to Hold Patient-Led Listening Session with FDA Read More »
Cure SMA is pleased to announce the launch of our newly updated merch store! The merch store features all the most popular Cure SMA t-shirts, hats, zip-up jackets, and more, as well as new items, which include: Black Performance Full-Zip Fleece Bucket Hat Women’s White Reflective Performance Half Zip Women’s Performance Racerback Tank In addition
Shop New Cure Merch at Our Updated Online Store Read More »
Thank you to everyone who attended the 2022 Annual SMA Conference at the Disneyland Hotel in Anaheim, California! It was an impactful weekend of opportunities to connect, learn, and have fun! We are happy to announce that, including on-site registration, there were over 2,100 attendees in total, which was a record for the Annual SMA
Thank You for a Wonderful 2022 Annual SMA Conference! Read More »
Cure SMA is pleased to announce the participation of scientific leadership in the 2022 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference (March 13–16, 2022), the American Academy of Neurology (AAN) 2022 Annual Meeting (April 2–7, 2022, and the upcoming Cure SMA Research & Clinical Care Meeting (June 15–17, 2022). Our poster presentations feature data from research
