A potential oral treatment for children with type 1 spinal muscular atrophy (SMA) called RG7916 was able to safely raise levels of the crucial SMN protein that is lacking in these patients, ... Read more
Ionis Pharmaceuticals has received $40 million in milestone earnings from Biogen following Japanese regulatory approval of the price of Spinraza, Biogen’s treatment for spinal muscular atrophy.
So far, Ionis has earned more ... Read more
Spinal muscular atrophy (SMA) is a genetic neuromuscular disease. SMA patients have either a missing or altered gene, (the survival motor neuron-1 or SMN1 gene), which causes large motor nerves ... Read more
To say that SMA can throw a curveball at you at any given moment is like saying that the “Star Wars” prequels could’ve benefited from more rewrites. There are ... Read more
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