To Fight High-Priced Drugs, Sidestep the Disease
Spinal muscular atrophy type 1 (SMA) begins when a parent notices that her infant can’t lift his head, a key milestone in early development. It ends with the silent passing of a toddler, his body lacking the neuromuscular function to live. This deadly disease affects almost 400 newborns in the United States each year, and approximately 13,000 per year globally. It arises because of a single defective gene. There is no cure.
In 2016, the FDA approved the first disease-modifying drug for SMA called nusinersen (Spinraza). It works by using a small…
