Gene Replacement Improves Survival in Spinal Muscular Atrophy
The treatment provides improvements in motor function that may be maintained for as long as two years.
Neurology Reviews. 2018 January;26(1):32
January 3, 2018|Breast Cancer ICYMI
—Erik Greb
Suggested Reading
Mendell JR, Al-Zaidy S, Shell R, et al. Single-dose gene-replacement therapy for spinal muscular atrophy. N Engl J Med. 2017;377(18):1713-1722.
van der Ploeg AT. The dilemma of two innovative therapies for spinal muscular atrophy. N Engl J Med. 2017;377(18):1786-1787.
