2017-11-02
The dilemma of two innovative therapies for spinal muscular atrophy
Publication
Publication
New England Journal of Medicine , Volume 377 - Issue 18 p. 1786- 1787
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 | |
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| doi.org/10.1056/NEJMe1712106, hdl.handle.net/1765/102726 | |
| New England Journal of Medicine | |
| Organisation | Department of Clinical Genetics |
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van der Ploeg, A. (2017). The dilemma of two innovative therapies for spinal muscular atrophy. New England Journal of Medicine, 377(18), 1786–1787. doi:10.1056/NEJMe1712106 |
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