For many people living with rare neuromuscular diseases, the most meaningful part of a recent Chicago gathering was not just the medical updates — it was finding a community that understood their daily struggles. The realization that they were not alone set the tone for MDA Engage: Chicago, a…
Discussion
Sleep-related breathing problems were found to ease over time in children with spinal muscular atrophy (SMA) types 2 and 3 receiving the widely approved therapies Evrysdi (risdiplam) or Spinraza (nusinersen). That’s according to a new study from Sweden that followed children and teens with the two disease…
Discussion
September always reminds me of the anniversaries of two incomprehensible disasters in the U.S.: 9/11 and Hurricane Helene, which devastated western North Carolina, including Ashe County, where I live. This year, though, September brought cause for celebration for people affected by spinal muscular atrophy (SMA). Earlier this month, the…
Discussion
“Alexa, turn off the alarm.” “OK. Good morning, Connie. Would you like to hear an inspirational quote?” “Sure, Alexa. Go for it.” “All right. ‘There is no elevator to success. You have to take the stairs.'” “But Alexa, I’m in a wheelchair.” “I cannot help you with that.” As a…
Discussion
Muscle regeneration, the benefits of a specialized diet, physiotherapy, and assistive technologies are among top priorities for spinal muscular atrophy (SMA) research identified by patients, caregivers, and healthcare professionals from 22 countries in Europe. The top 10 priorities, developed through a series of surveys and a workshop, were published…
Discussion
The brain has long been thought of as a singular organ, but a new study shows that the front and back parts of the brain actually develop completely independently of each other. This discovery may have key implications for research into diseases like spinal muscular atrophy (SMA), which affects…
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