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Novel challenges in spinal muscular atrophy – How to screen and whom to treat?

In recent years, disease‐modifying and life‐prolonging therapies for spinal muscular atrophy (SMA) have been developed. However, patients are currently diagnosed with significant delay and therapies are often administered in advanced stages of motor neuron degeneration, showing limited effects. Meth...

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Bibliographic Details
Published in:Ann Clin Transl Neurol
Main Authors: Saffari, Afshin, Kölker, Stefan, Hoffmann, Georg F., Weiler, Markus, Ziegler, Andreas
Format: Artigo
Language:Inglês
Published: John Wiley and Sons Inc. 2018
Subjects:
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC6331314/
https://ncbi.nlm.nih.gov/pubmed/30656198
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/acn3.689
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