it helps some kids gain some strength, some who take the drug still die (49% in the initial trial showed no improvement)
It's perhaps the most impressive medication I've seen developed. Kids with SMA type 1 typically die in the first year of life, and they often never develop enough strength to even sit. There has never been any treatment, or anything close to a treatment, for SMA. Kids with SMA don't make SMN protein, which is needed for normal nerve function. Spinraza, through genetic manipulation, tricks the body into making SMN protein. Kids treated with Spinraza have sat and even walked when they otherwise would not have been expected to (the studies have only followed about 1 year of treatment). 40% of kids treated with Spinraza gained motor milestones, whereas 0% did in the placebo group. The results are incredibly impressive.