Cure SMA Offers Free Virtual Therapy Sessions
To help with stress or anxiety brought on by the COVID-19 pandemic or other issues, adult patients with spinal muscular atrophy (SMA) in the U.S. are ... Read more
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This Rare Disease Day, I’m Grateful for My Friends
You know it’s a unique relationship when one friend helps the other use the bathroom. This is the scenario I found myself in a couple ... Read more -
Rare Disease Day at NIH, Set for March 1, Growing Year by Year
Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, research ... Read more -
How a Head Cold Saved My Life (Figuratively)
We all know that I hate getting up early. Just like I hate adjusting to a new wheelchair — I’ll do it if I have ... Read more
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Researchers Discover Promising New Gene Therapy Approach for Spinal Muscular Atrophy
News
A new genetic approach to restore the expression of the gene that causes spinal muscular atrophy (SMA) in patients’ cells holds promise to effectively and permanently treat the disease, researchers suggest. The study, “Seamless genetic conversion of SMN2 to SMN1 ... Read more