The therapy is "life changing
for those individuals who receive a benefit from this." They go "from
darkness" and perhaps being "dependent on others" to gaining
"independence, mobility and a significant increase in quality of
life....The juice is worth the squeeze."
— Stephen M. Rose, Ph.D., chief
research officer at the Foundation Fighting Blindness, tells AIS Health about
Spark Therapeutics Inc.'s Luxturna (voretigene neparvovec), which if approved
by the FDA would be the first therapy where a corrective gene is given to a
patient. The drug, which treats a condition that causes blindness, also may be
the first therapy in the United States to top $1 million for a single patient.
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