The dilemma of two innovative therapies for spinal muscular atrophy
If you have made a diagnosis of spinal muscular atrophy (SMA) type 1 (also known as Werdnig–Hoffman disease) in a child, then you have vivid memories of informing desperate parents that there is nothing you can do for their child. Two innovative therapies for SMA may now bring some hope — but what do they mean for patients and their families?
|Persistent URL||dx.doi.org/10.1056/NEJMe1712106, hdl.handle.net/1765/102726|
|Journal||New England Journal of Medicine|
van der Ploeg, A.T. (2017). The dilemma of two innovative therapies for spinal muscular atrophy. New England Journal of Medicine, 377(18), 1786–1787. doi:10.1056/NEJMe1712106