This Rare Disease Day, I’m Grateful for My FriendsYou 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 YearRare 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
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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