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?

Additional Metadata
Persistent URL dx.doi.org/10.1056/NEJMe1712106, hdl.handle.net/1765/102726
Journal New England Journal of Medicine
Note No subscription
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