The FDA recently approved Spinraza, the first drug designed to treat spinal muscular atrophy. According to a press release from the manufacturer, infants with SMA who received the drug during clinical trials were much more likely to show improvements in motor function than those left untreated. Spinraza is approved to treat both children and adults with SMA.
It’s a bit surreal to live in a time where a specific treatment exists for my disability. I have no idea whether the drug would personally benefit me and I’m not in any hurry to find out. After living with this disability for four decades, my body’s remaining muscle tissue probably resembles the gristle of a cheap steak and I’m not sure any of it can be salvaged. This is probably thrilling news for parents with young children with SMA. It’s entirely possible that those kids will live to see a day when genetic therapies can effectively manage or even cure their condition. In another forty years, people like me could be a historical curiosity, invoking the same reactions that I had when I first saw pictures of kids living in iron lungs in the 1950s:
“People really lived like that?”